Trial Outcomes & Findings for A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis (NCT NCT03549871)
NCT ID: NCT03549871
Last Updated: 2023-02-06
Results Overview
Bleeding episodes (BE): any occurrence of hemorrhage might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours of last injection at that location was considered as a new event. ABR = total number of qualifying BE/total number of days in the respective period\*365.25. Estimated data were derived by using repeated measures negative binomial (NB) regression model.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
80 participants
Primary outcome timeframe
Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
Results posted on
2023-02-06
Participant Flow
Study was conducted at 35 active sites in 15 countries. Total of 99 participants were screened from 25 July 2018 to 19 March 2021, of which 19 were screen failure due to not meeting eligibility criteria. Study had 2 main periods: 6-month factor/bypassing agent (BPA) prophylaxis period \& 7-month fitusiran treatment period (1-month onset and 6-month fitusiran efficacy period).
Subgroup of Cohort A: Participants with hemophilia B with inhibitors and inadequate response to BPA treatment (historical annualized bleeding rate \[ABR\] greater than or equal to \[\>=\] 20) prior to enrollment did not participate in 6-month prophylaxis period and they entered directly into fitusiran treatment period.
Participant milestones
| Measure |
Cohort A: Inhibitor
Participants with hemophilia A or B and inhibitory antibodies to coagulation factor VIII (FVIII) or coagulation factor IX (FIX) and who were receiving BPA prophylaxis were enrolled in the study and entered into 6-month factor or BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with BPAs (twice weekly \[activated prothrombin complex concentrates {aPCC}\] or every other day \[recombinant factor VIIa {rFVIIa}\]). This period was skipped by a subgroup of Cohort A (hemophilia B with inhibitors and historical ABR \>= 20) and they entered directly in to fitusiran treatment period. Post completion of BPA prophylaxis period, participants entered 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 milligrams (mg) once monthly (QM) as subcutaneous (SC) injection for 7 months until the sponsor initiated a voluntary dose pause. After dose pause and protocol amendment, the fitusiran dose was changed to 50 mg once every other month (Q2M) as SC injection. Participants could continue to receive BPA prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout study, participants in fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs, as appropriate.
|
Cohort B: Non-inhibitor
Participants with hemophilia A or B, without inhibitory antibodies to FVIII or FIX who were receiving factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with factor concentrates (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]). Post completion of factor prophylaxis period, participants entered in to 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection until the sponsor initiated a voluntary dose pause. Participants could continue to receive their factor prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with factor, as appropriate.
|
|---|---|---|
|
Factor/BPA Prophylaxis: Day -168 to -1
STARTED
|
30
|
50
|
|
Factor/BPA Prophylaxis: Day -168 to -1
Started Directly With Fitusiran
|
2
|
0
|
|
Factor/BPA Prophylaxis: Day -168 to -1
Started With Factor or BPA Prophylaxis
|
28
|
50
|
|
Factor/BPA Prophylaxis: Day -168 to -1
COMPLETED
|
23
|
46
|
|
Factor/BPA Prophylaxis: Day -168 to -1
NOT COMPLETED
|
7
|
4
|
|
Fitusiran Treatment: Day 1 to Day 190
STARTED
|
23
|
46
|
|
Fitusiran Treatment: Day 1 to Day 190
Received 80mg QM & Part of Safety Analysis Set 1 (SAS 1)
|
21
|
46
|
|
Fitusiran Treatment: Day 1 to Day 190
Received 50 mg Q2M & Part of Safety Analysis Set 2 (SAS 2)
|
2
|
0
|
|
Fitusiran Treatment: Day 1 to Day 190
Efficacy Analysis Set 1 (EAS 1)
|
19
|
46
|
|
Fitusiran Treatment: Day 1 to Day 190
Efficacy Analysis Set 2 (EAS 2)
|
2
|
0
|
|
Fitusiran Treatment: Day 1 to Day 190
COMPLETED
|
17
|
37
|
|
Fitusiran Treatment: Day 1 to Day 190
NOT COMPLETED
|
6
|
9
|
Reasons for withdrawal
| Measure |
Cohort A: Inhibitor
Participants with hemophilia A or B and inhibitory antibodies to coagulation factor VIII (FVIII) or coagulation factor IX (FIX) and who were receiving BPA prophylaxis were enrolled in the study and entered into 6-month factor or BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with BPAs (twice weekly \[activated prothrombin complex concentrates {aPCC}\] or every other day \[recombinant factor VIIa {rFVIIa}\]). This period was skipped by a subgroup of Cohort A (hemophilia B with inhibitors and historical ABR \>= 20) and they entered directly in to fitusiran treatment period. Post completion of BPA prophylaxis period, participants entered 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 milligrams (mg) once monthly (QM) as subcutaneous (SC) injection for 7 months until the sponsor initiated a voluntary dose pause. After dose pause and protocol amendment, the fitusiran dose was changed to 50 mg once every other month (Q2M) as SC injection. Participants could continue to receive BPA prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout study, participants in fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs, as appropriate.
|
Cohort B: Non-inhibitor
Participants with hemophilia A or B, without inhibitory antibodies to FVIII or FIX who were receiving factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with factor concentrates (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]). Post completion of factor prophylaxis period, participants entered in to 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection until the sponsor initiated a voluntary dose pause. Participants could continue to receive their factor prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with factor, as appropriate.
|
|---|---|---|
|
Factor/BPA Prophylaxis: Day -168 to -1
Withdrawal by Subject
|
0
|
2
|
|
Factor/BPA Prophylaxis: Day -168 to -1
Adverse Event
|
1
|
0
|
|
Factor/BPA Prophylaxis: Day -168 to -1
Parent withdrew consent
|
1
|
0
|
|
Factor/BPA Prophylaxis: Day -168 to -1
Switched to treatment not per protocol
|
4
|
0
|
|
Factor/BPA Prophylaxis: Day -168 to -1
Related to Coronavrus Disease 2019
|
1
|
0
|
|
Factor/BPA Prophylaxis: Day -168 to -1
Did not meet trial eligibility
|
0
|
2
|
|
Fitusiran Treatment: Day 1 to Day 190
Withdrawal by Subject
|
1
|
0
|
|
Fitusiran Treatment: Day 1 to Day 190
More than 1 antithrombin (AT) measurement less than 15%
|
1
|
0
|
|
Fitusiran Treatment: Day 1 to Day 190
Study drug on hold
|
4
|
5
|
|
Fitusiran Treatment: Day 1 to Day 190
Participant's decision
|
0
|
2
|
|
Fitusiran Treatment: Day 1 to Day 190
Adverse Event
|
0
|
2
|
Baseline Characteristics
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Baseline characteristics by cohort
| Measure |
Cohort A: Inhibitor
n=23 Participants
Participants with hemophilia A or B and inhibitory antibodies to FVIII or FIX and who were receiving BPA prophylaxis were enrolled in the study and entered into 6-month factor or BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]). This period was skipped by a subgroup of Cohort A (hemophilia B with inhibitors and historical ABR \>= 20) and they entered directly in to fitusiran treatment period. Post completion of BPA prophylaxis period, participants entered 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection for 7 months until the sponsor initiated a voluntary dose pause. After dose pause and protocol amendment, the fitusiran dose was changed to 50 mg Q2M as SC injection. Participants could continue to receive BPA prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout the study, participants in fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs, as appropriate.
|
Cohort B: Non-inhibitor
n=46 Participants
Participants with hemophilia A or B, without inhibitory antibodies to FVIII or FIX who were receiving factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with factor concentrates (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]). Post completion of factor prophylaxis period, participants entered in to 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection until the sponsor initiated a voluntary dose pause. Participants could continue to receive their factor prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with factor, as appropriate.
|
Total Title
n=69 Participants
|
|---|---|---|---|
|
Age, Continuous
|
27.7 years
STANDARD_DEVIATION 15.9 • n=5 Participants
|
23.5 years
STANDARD_DEVIATION 7.3 • n=7 Participants
|
24.9 years
STANDARD_DEVIATION 11.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
69 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=5 Participants
|
27 Participants
n=7 Participants
|
45 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on efficacy analysis set 1 (EAS1) which included participants who received factor/BPA prophylaxis and any dose of fitusiran before dose resumption. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
Bleeding episodes (BE): any occurrence of hemorrhage might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours of last injection at that location was considered as a new event. ABR = total number of qualifying BE/total number of days in the respective period\*365.25. Estimated data were derived by using repeated measures negative binomial (NB) regression model.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=65 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Estimated Annualized Bleeding Rate (ABR)
|
7.482 episodes per participant per year
Interval 5.52 to 10.141
|
2.908 episodes per participant per year
Interval 1.727 to 4.898
|
PRIMARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS1 population. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
A bleeding episode (BE): any occurrence of hemorrhage might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original BE and counted as 1 BE towards ABR. Any bleeding that began after 72 hours of last injection at that location was considered as a new event. ABR = total number of qualifying BE/number of days in the respective period \*365.25.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=65 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Observed Annualized Bleeding Rate (ABR)
|
7.56 episodes per participant per year
Standard Deviation 9.49
|
3.19 episodes per participant per year
Standard Deviation 7.75
|
SECONDARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
BE: any occurrence of hemorrhage that might require administration of factor/BPA infusion. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours of last injection at that location was considered as a new event. Spontaneous BE: BE occurrence for no apparent/known reason, particularly into joints, muscles, and soft tissues. ABR = total number of qualifying BE/number of days in respective period \*365.25. Estimated data was derived using repeated measures NB regression model.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=65 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Estimated Annualized Spontaneous Bleeding Rate
|
5.002 episodes per participant per year
Interval 3.424 to 7.305
|
2.222 episodes per participant per year
Interval 1.19 to 4.152
|
SECONDARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and was counted as 1 BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as a new event. Spontaneous BE: bleeding event occurred for no apparent or known reason, particularly into joints, muscles and soft tissues. ABR = total number of qualifying BE/number of days in respective period \*365.25.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=65 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Observed Annualized Spontaneous Bleeding Rate
|
5.09 episodes per participant per year
Standard Deviation 7.93
|
2.51 episodes per participant per year
Standard Deviation 7.33
|
SECONDARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
BE: any hemorrhage that required administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection to treat BE at that location was considered part of original BE; counted as 1 BE towards ABR. Bleeding after 72 hours from last injection at that location was considered as a new event. Joint BE: characterized by unusual sensation in joint ("aura") + increasing swelling/warmth over joint skin, increasing pain/progressive loss of range of motion/difficulty in limb use compared to Baseline. ABR = total number of qualifying BE/number of days in respective period \*365.25. Estimated data were derived by using repeated measures NB regression model.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=65 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Estimated Annualized Joint Bleeding Rate
|
5.282 episodes per participant per year
Interval 3.647 to 7.651
|
2.564 episodes per participant per year
Interval 1.44 to 4.566
|
SECONDARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as a new event. Joint BE: characterized by unusual sensation in joint (aura) increasing swelling/warmth over joint skin, increasing pain or progressive loss of range of motion/difficulty in limb use compared to Baseline. ABR = total number of joint BE/number of days in respective period\*365.25.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=65 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Observed Annualized Joint Bleeding Rate
|
5.35 episodes per participant per year
Standard Deviation 8.19
|
2.82 episodes per participant per year
Standard Deviation 7.54
|
SECONDARY outcome
Timeframe: Month -6 of Factor or BPA prophylaxis period (Baseline), Day 1 (Month 1) and Month 7 of fitusiran treatment periodPopulation: Analysis was performed on EAS 1 population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
Haem-A-QoL: participant-reported questionnaire for adults aged \>=17 years with hemophilia and comprised of 46 items covering 10 domains. Physical health domain (PHD) is assessed with 5 items rated on 5-point Likert scale: never, rarely, sometimes, often or all the time. Raw score for PHD were transformed to scale ranged from 0 to 100, where lower scores = better physical health. Least square (LS) mean and 95% confidence interval (CI) by mixed model for repeated measure (MMRM) analysis with robust sandwich covariance matrix: change from Month -6 to Day 1 and to Month 7 as response variable; period (factor/BPA prophylaxis \& fitusiran treatment) \& Baseline score (Month -6) as fixed effects.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=48 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=48 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Change in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Score in the Fitusiran Treatment Period and the Factor or BPA Prophylaxis Period
|
-6.00 score on a scale
Interval -10.19 to -1.81
|
-9.60 score on a scale
Interval -15.35 to -3.84
|
SECONDARY outcome
Timeframe: Month -6 of Factor or BPA prophylaxis period (Baseline), Day 1 (Month 1) and Month 7 of fitusiran treatment periodPopulation: Analysis was performed on EAS1 population. Here, 'overall number of participants analyzed = participants with available data for this outcome measure. Data collection and analysis of combined population of Cohort A and B for each period was planned and performed for this outcome measure.
Haem-A-QoL: questionnaire for adults aged \>= 17 years with hemophilia; and comprised of 46 items covering 10 domains: physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality. Items were rated on 5-point Likert scale: never, rarely, sometimes, often or all time. Domain raw score was transformed to scale ranged from 0 to 100, where lower scores=better health. LS mean \& 95% CI by MMRM analysis with robust sandwich covariance matrix: change from Month -6 to Day 1 and to Month 7 as response variable; period (factor/BPA prophylaxis \& fitusiran treatment) \& Baseline score (Month -6) as fixed effects.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=48 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=48 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Change in Haemophilia Quality of Life Questionnaire for Adults Total Score in the Fitusiran Treatment Period and the Factor or BPA Prophylaxis Period
|
-3.07 score on a scale
Interval -5.56 to -0.58
|
-7.62 score on a scale
Interval -10.26 to -4.98
|
SECONDARY outcome
Timeframe: Day 1 up to Day 28 or up to the last day of bleeding follow up (any day up to Day 28), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Data collection and analysis of combined population of Cohort A and B for fitusiran 1-month onset period was planned and performed for this outcome measure.
BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and was counted as 1 BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as new event. Estimated ABR and 95% CI was derived by using repeated measures NB regression model with logarithm of duration (years) that each participant spends in 1-Month fitusiran onset period matching BE data being analyzed as offset variable. ABR = total number of qualifying BE/number of days in respective period \*365.25.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Estimated Annualized Bleeding Rate in the Fitusiran Onset Period
|
5.419 episodes per participant per year
Interval 3.716 to 7.901
|
—
|
SECONDARY outcome
Timeframe: Day 1 up to Day 28 or up to the last day of bleeding follow up (any day up to Day 28), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Data collection and analysis of combined population of Cohort A and B for fitusiran 1-month onset period was planned and performed for this outcome measure.
BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original BE and was counted as one BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as a new event. ABR= total number of qualifying BE/number of days in the 1-month onset period \*365.25. Analysis was based on on-treatment strategy which included all treated bleeding events in 1-month onset period and excluded any bleeding events in period of intercurrent events.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Observed Annualized Bleeding Rate in the Fitusiran Onset Period
|
5.42 episodes per participant per year
Standard Deviation 8.28
|
—
|
SECONDARY outcome
Timeframe: From Day 1 up to Day 190Population: Analysis was performed on EAS 1 population. Data collection and analysis of combined population of Cohort A and B for fitusiran treatment period was planned and performed for this outcome measure.
BE: defined as any occurrence of hemorrhage that might require administration of factor/BPA. BE start time was time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original BE and counted as one BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as new event. Analysis was based on on-treatment strategy which included all treated bleeding events in fitusiran period and excluded any bleeding events in the period of intercurrent events. ABR = total number of qualifying BE/number of days in respective period \*365.25.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Estimated Annualized Bleeding Rate in the Fitusiran Treatment Period
|
3.317 episodes per participant per year
Interval 2.111 to 5.211
|
—
|
SECONDARY outcome
Timeframe: from Day 1 up to Day 190Population: Analysis was performed on EAS1 population. Data collection and analysis of combined population of Cohort A and B for fitusiran treatment period was planned and performed for this outcome measure.
BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time was time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original bleeding event and was counted as one BE towards ABR. Any bleeding that began after 72 hours from last injection at that location was considered as a new event. ABR= total number of qualifying BE/number of days in treatment period \*365.25. Analysis was based on on-treatment strategy which included all treated bleeding events in fitusiran period and excluded any bleeding events in period of intercurrent events.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=65 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Observed Annualized Bleeding Rate in the Fitusiran Treatment Period
|
3.48 episodes per participant per year
Standard Deviation 6.98
|
—
|
SECONDARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure. Combined data of annualized weight-adjusted BPA consumption (U/kg) for both treated bleeds and prophylaxis purpose were reported in this outcome measure.
Annualized weight-adjusted BPA consumption was calculated for each participant during prophylaxis period as: \[Sum of BPA dose per body weight received during corresponding period/number of days in corresponding period\]\*365.25. In this outcome measure, data of annualized weight-adjusted consumption of BPA agent: aPCC (unit per kilogram \[U/kg\]) were reported.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=15 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=15 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Cohort A: Annualized Weight-adjusted Consumption of BPA (Activated Prothrombin Complex Concentrates)
|
7912.7 U/kg per participant per year
Standard Deviation 5507.6
|
39.7 U/kg per participant per year
Standard Deviation 87.0
|
SECONDARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure. Combined data of annualized weight-adjusted BPA consumption (mcg/kg) for both treated bleeds and prophylaxis purpose were reported in this outcome measure.
Annualized weight-adjusted BPA consumption was calculated for each participant during prophylaxis period as: (Sum of BPA dose per body weight received during corresponding period/number of days in corresponding period)\*365.25. In this outcome measure, data of annualized weight-adjusted consumption of BPA agents: rFVIIa (unit: micrograms per kg \[mcg/kg\]) were reported. Combined data of annualized weight-adjusted BPA consumption (mcg/kg) for both treated bleeds and prophylaxis purpose were reported in this outcome measure.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=7 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=7 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Cohort A: Annualized Weight-adjusted Consumption of BPA (Recombinant Factor VIIa)
|
18895.8 mcg/kg per participant per year
Standard Deviation 14081.9
|
168.8 mcg/kg per participant per year
Standard Deviation 290.8
|
SECONDARY outcome
Timeframe: Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliestPopulation: Analysis was performed on EAS 1 population. Here, 'number analyzed' = participants with available data for each specified category. Combined data of annualized weight-adjusted factor consumption (IU/kg) for both treated bleeds and prophylaxis purpose were reported in this outcome measure.
Annualized weight-adjusted BPA consumption was calculated for each participant during prophylaxis period as: (Sum of BPA dose per body weight received during corresponding period/number of days in corresponding period)\*365.25. In this outcome measure, data of annualized weight-adjusted consumption of BPA agents: FVIII and FIX (unit: international Units \[IU\] per kg \[IU/kg\]) were reported.
Outcome measures
| Measure |
Overall Factor/BPA Prophylaxis Period
n=46 Participants
All participants, whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII\], once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall Fitusiran 80 mg Efficacy Period
n=46 Participants
All participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause during fitusiran efficacy period (i.e., from Day 29 to Day 190 of fitusiran treatment period) and were considered for efficacy analysis during fitusiran efficacy period.
|
|---|---|---|
|
Cohort B: Annualized Weight-adjusted Consumption of Factor
FVIII
|
3396.9 IU/kg per participant per year
Standard Deviation 1144.5
|
60.7 IU/kg per participant per year
Standard Deviation 148.3
|
|
Cohort B: Annualized Weight-adjusted Consumption of Factor
FIX
|
3175.5 IU/kg per participant per year
Standard Deviation 961.3
|
17.8 IU/kg per participant per year
Standard Deviation 56.1
|
Adverse Events
Cohort A: SAS 1 - BPA Prophylaxis
Serious events: 5 serious events
Other events: 10 other events
Deaths: 0 deaths
Cohort A: SAS 1 - Fitusiran 80 mg QM
Serious events: 5 serious events
Other events: 13 other events
Deaths: 0 deaths
Cohort B: SAS 1 - Factor Prophylaxis
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Cohort B: SAS 1 - Fitusiran 80 mg QM
Serious events: 4 serious events
Other events: 28 other events
Deaths: 0 deaths
Overall: SAS 1 - Factor/BPA Prophylaxis
Serious events: 5 serious events
Other events: 19 other events
Deaths: 0 deaths
Overall: SAS 1 - Fitusiran 80 mg QM
Serious events: 9 serious events
Other events: 41 other events
Deaths: 0 deaths
Cohort A: SAS 2 - BPA Prophylaxis
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort A: SAS 2 - Fitusiran 50 mg Q2M
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort A: SAS 1 - BPA Prophylaxis
n=19 participants at risk
Participants with hemophilia A or B, with inhibitory antibodies to FVIII or FIX and who were receiving BPA prophylaxis were enrolled in the study and entered the 6-month BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]).
|
Cohort A: SAS 1 - Fitusiran 80 mg QM
n=21 participants at risk
Post completion of BPA prophylaxis period, participants entered into the 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection until sponsor initiated a voluntary dose pause. Participants could continue to receive BPA prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. A subgroup of Cohort A (participants with hemophilia B and inhibitory antibodies to FIX who had not responded adequately to BPA prophylaxis prior to study entry \[historical ABR \>=20\]) entered directly into fitusiran treatment period. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs, as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
Cohort B: SAS 1 - Factor Prophylaxis
n=46 participants at risk
Participants with hemophilia A or B, without inhibitory antibodies to FVIII or FIX and who were receiving factor prophylaxis were enrolled in the study and entered the 6-month BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with factor concentrates (twice weekly \[standard half-life FVIII), once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]).
|
Cohort B: SAS 1 - Fitusiran 80 mg QM
n=46 participants at risk
Post completion of Factor prophylaxis period, participants entered into the 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection until sponsor initiated a voluntary dose pause (up to maximum of 7 months). Participants could continue to receive BPA prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with factor, as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
Overall: SAS 1 - Factor/BPA Prophylaxis
n=65 participants at risk
All participants whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis, respectively, were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII), once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall: SAS 1 - Fitusiran 80 mg QM
n=67 participants at risk
Post completion of BPA prophylaxis period, all participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause (up to maximum of 7 months). Participants could continue to receive BPA/factor prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. A subgroup of Cohort A (participants with hemophilia B and inhibitory antibodies to FIX who had not responded adequately to BPA prophylaxis treatment \[historical ABR \>=20\]) entered directly into fitusiran treatment period. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs or factors, as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
Cohort A: SAS 2 - BPA Prophylaxis
n=2 participants at risk
Participants with hemophilia A or B, with inhibitory antibodies to FVIII or FIX and who were receiving BPA prophylaxis were enrolled in the study and entered the 6-month BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]).
|
Cohort A: SAS 2 - Fitusiran 50 mg Q2M
n=2 participants at risk
Post completion of BPA prophylaxis period, participants entered into the 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 50 mg Q2M as SC injection after sponsor initiated voluntary dose pause and protocol amendment. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]), as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
|---|---|---|---|---|---|---|---|---|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Injury, poisoning and procedural complications
Femur Fracture
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Investigations
C-Reactive Protein Increased
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Nervous system disorders
Cerebrovascular Accident
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Pancreatitis Acute
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Skin and subcutaneous tissue disorders
Stevens-Johnson Syndrome
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
50.0%
1/2 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Musculoskeletal and connective tissue disorders
Haemophilic Arthropathy
|
10.5%
2/19 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.1%
2/65 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.0%
2/67 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Musculoskeletal and connective tissue disorders
Muscle Haemorrhage
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Covid-19 Pneumonia
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
50.0%
1/2 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Gastroenteritis
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Vascular Device Infection
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Surgical and medical procedures
Central Venous Catheter Removal
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Biliary Neoplasm
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Respiratory, thoracic and mediastinal disorders
Asthma Late Onset
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
Other adverse events
| Measure |
Cohort A: SAS 1 - BPA Prophylaxis
n=19 participants at risk
Participants with hemophilia A or B, with inhibitory antibodies to FVIII or FIX and who were receiving BPA prophylaxis were enrolled in the study and entered the 6-month BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]).
|
Cohort A: SAS 1 - Fitusiran 80 mg QM
n=21 participants at risk
Post completion of BPA prophylaxis period, participants entered into the 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection until sponsor initiated a voluntary dose pause. Participants could continue to receive BPA prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. A subgroup of Cohort A (participants with hemophilia B and inhibitory antibodies to FIX who had not responded adequately to BPA prophylaxis prior to study entry \[historical ABR \>=20\]) entered directly into fitusiran treatment period. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs, as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
Cohort B: SAS 1 - Factor Prophylaxis
n=46 participants at risk
Participants with hemophilia A or B, without inhibitory antibodies to FVIII or FIX and who were receiving factor prophylaxis were enrolled in the study and entered the 6-month BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with factor concentrates (twice weekly \[standard half-life FVIII), once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]).
|
Cohort B: SAS 1 - Fitusiran 80 mg QM
n=46 participants at risk
Post completion of Factor prophylaxis period, participants entered into the 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 80 mg QM as SC injection until sponsor initiated a voluntary dose pause (up to maximum of 7 months). Participants could continue to receive BPA prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with factor, as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
Overall: SAS 1 - Factor/BPA Prophylaxis
n=65 participants at risk
All participants whether with inhibitory antibodies (Cohort A) or without inhibitory antibodies (Cohort B) to FVIII or FIX and who were receiving BPA or factor prophylaxis, respectively, were enrolled in the study and entered the 6-month factor or BPA prophylaxis period in this study and continued their pre-study regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]) or factors (twice weekly \[standard half-life FVIII), once weekly \[extended half-life FVIII; standard half-life FIX\], and once biweekly \[extended half-life FIX\]) in 6-month factor or BPA prophylaxis period. This period was skipped by a subgroup of Cohort A which included participants with hemophilia B with inhibitory antibodies to FIX and who had not responded adequately to BPA prophylaxis prior to study entry (historical ABR \>=20).
|
Overall: SAS 1 - Fitusiran 80 mg QM
n=67 participants at risk
Post completion of BPA prophylaxis period, all participants who received fitusiran 80 mg QM as SC injection (either in Cohort A or Cohort B) until sponsor initiated a voluntary dose pause (up to maximum of 7 months). Participants could continue to receive BPA/factor prophylaxis for up to 7 days after start of fitusiran treatment on Day 1. A subgroup of Cohort A (participants with hemophilia B and inhibitory antibodies to FIX who had not responded adequately to BPA prophylaxis treatment \[historical ABR \>=20\]) entered directly into fitusiran treatment period. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs or factors, as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
Cohort A: SAS 2 - BPA Prophylaxis
n=2 participants at risk
Participants with hemophilia A or B, with inhibitory antibodies to FVIII or FIX and who were receiving BPA prophylaxis were enrolled in the study and entered the 6-month BPA prophylaxis period in this study and continued their pre-study, regularly scheduled prophylaxis regimen with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]).
|
Cohort A: SAS 2 - Fitusiran 50 mg Q2M
n=2 participants at risk
Post completion of BPA prophylaxis period, participants entered into the 7-month fitusiran treatment period (1-month onset period + 6-month efficacy period) and received fitusiran 50 mg Q2M as SC injection after sponsor initiated voluntary dose pause and protocol amendment. Throughout the study, participants in the fitusiran treatment period could receive on-demand treatment for breakthrough bleeding episodes with BPAs (twice weekly \[aPCC\] or every other day \[rFVIIa\]), as appropriate. Post last fitusiran dose, participants had a safety follow-up/AT activity level follow-up up to 6 months (monthly monitor until AT activity levels return to approximately 60% or per Investigator discretion).
|
|---|---|---|---|---|---|---|---|---|
|
Injury, poisoning and procedural complications
Arthropod Bite
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Injury, poisoning and procedural complications
Buttock Injury
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Injury, poisoning and procedural complications
Fall
|
10.5%
2/19 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.1%
2/65 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Investigations
Alanine Aminotransferase Increased
|
5.3%
1/19 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
14.3%
3/21 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
32.6%
15/46 • Number of events 19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
26.9%
18/67 • Number of events 22 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Investigations
Aspartate Aminotransferase Increased
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.5%
3/46 • Number of events 4 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.0%
4/67 • Number of events 7 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Investigations
Fibrin D Dimer Increased
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
14.3%
3/21 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.3%
2/46 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
7.5%
5/67 • Number of events 5 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
50.0%
1/2 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Investigations
Lymphocyte Count Increased
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
50.0%
1/2 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Investigations
Neutrophil Count Decreased
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
50.0%
1/2 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Investigations
White Blood Cell Count Decreased
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
50.0%
1/2 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Blood and lymphatic system disorders
Anaemia
|
10.5%
2/19 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.1%
2/65 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Nervous system disorders
Headache
|
10.5%
2/19 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.6%
3/65 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.5%
2/21 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.5%
3/67 • Number of events 4 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Dental Caries
|
5.3%
1/19 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.1%
2/65 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Eosinophilic Oesophagitis
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Gastrointestinal Motility Disorder
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
50.0%
1/2 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Nausea
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Teething
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.0%
2/67 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
100.0%
2/2 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Gastrointestinal disorders
Vomiting
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Skin and subcutaneous tissue disorders
Dermatitis Allergic
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.5%
3/46 • Number of events 4 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
8.7%
4/46 • Number of events 8 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.2%
4/65 • Number of events 5 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
7.5%
5/67 • Number of events 9 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Musculoskeletal and connective tissue disorders
Joint Swelling
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Musculoskeletal and connective tissue disorders
Synovitis
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/67 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Metabolism and nutrition disorders
Iron Deficiency
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Genital Herpes Simplex
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Influenza
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.3%
2/46 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.6%
3/65 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.0%
2/67 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.8%
1/21 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
15.2%
7/46 • Number of events 9 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
11.9%
8/67 • Number of events 10 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Tinea Pedis
|
5.3%
1/19 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/21 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
3.1%
2/65 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/67 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
15.8%
3/19 • Number of events 4 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
19.0%
4/21 • Number of events 5 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.3%
2/46 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.2%
4/65 • Number of events 5 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.0%
6/67 • Number of events 7 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
General disorders
Injection Site Erythema
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.5%
2/21 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.5%
3/67 • Number of events 4 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
General disorders
Injection Site Pain
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.5%
2/21 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.5%
3/46 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
7.5%
5/67 • Number of events 5 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.5%
2/21 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.3%
2/46 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.0%
4/67 • Number of events 5 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.5%
2/21 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.3%
2/46 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.0%
4/67 • Number of events 4 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.5%
2/21 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/46 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
6.5%
3/46 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/65 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
7.5%
5/67 • Number of events 5 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
|
Hepatobiliary disorders
Hepatic Steatosis
|
0.00%
0/19 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
9.5%
2/21 • Number of events 2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
2.2%
1/46 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
1.5%
1/65 • Number of events 1 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
4.5%
3/67 • Number of events 3 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
0.00%
0/2 • Factor/BPA prophylaxis period: from Day -168 to Day -1; Fitusiran 80 mg QM/50 mg Q2M period: from Day 1 of fitusiran treatment period (i.e., 1 month onset+ 6 months efficacy) until AT follow up (i.e. up to 21 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Safety analysis set (SAS): all participants who were enrolled and received any dose of fitusiran (80 mg, QM before dose pause; SAS 1)/(50 mg, Q2M after dose pause; SAS 2).
|
Additional Information
Trial Transparency Team
Sanofi ( Genzyme, a Sanofi Company )
Phone: 800-633-1610
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
- Publication restrictions are in place
Restriction type: OTHER