Trial Outcomes & Findings for Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (NCT NCT04323098)
NCT ID: NCT04323098
Last Updated: 2025-10-08
Results Overview
The change from baseline (assuming no treatment for severe hemophilia A) in FVIII activity, as measured by chromogenic substrate assay (CSA), at Week 52 (during Weeks 49 - 52) post-BMN 270 infusion. Each participant's FVIII activity level at Week 52 is defined as the median of the values obtained within the analysis window at Weeks 49-52. The baseline value will be imputed as 1 IU/dL, since there will be no washout of severe hemophilia A participants' usual FVIII prophylaxis (in order to avoid increasing the risk of bleeding) prior to BMN 270 infusion. Post-BMN 270 infusion values for FVIII activity will be excluded from analysis if obtained within 72 hours (or 3 calendar days if time is not available) since the last infusion of exogenous FVIII replacement therapy. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis.
COMPLETED
PHASE3
22 participants
Baseline to Week 52
2025-10-08
Participant Flow
This study was conducted by 12 principal investigators at 12 study centers in 4 countries (United States, Australia, Brazil, and Taiwan)
Total of 34 subjects were screened. Of these, 12 subjects failed screening and remaining 22 subject was enrolled in the study. Of the 22 enrolled participants, all 22 completed the Week 52 visit, and all 22 remained enrolled in the study as on data cut-off date 27January2023. While up to 25% of patients could be AAV5+, no such patients were enrolled, i.e., everyone was AAV5 negative.
Participant milestones
| Measure |
BMN 270 (valoctocogene roxaparvovec)
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, with Prophylactic Corticosteroids in Hemophilia A subjects
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
Continuing in the study
|
22
|
|
Overall Study
Completed Week 52 visit
|
22
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
22
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
n(%)
Baseline characteristics by cohort
| Measure |
BMN 270 (Valoctocogene Roxaparvovec)
n=22 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, with Prophylactic Corticosteroids in Hemophilia A subjects
|
|---|---|
|
Age, Continuous
|
28.0 years
STANDARD_DEVIATION 7.4 • n=5 Participants
|
|
Age, Customized
18 to < 30 years
|
13 Participants
n=5 Participants
|
|
Age, Customized
30 to < 50 years
|
9 Participants
n=5 Participants
|
|
Age, Customized
>= 50 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African-American
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
18 Participants
n=5 Participants
|
|
Baseline annualized FVIII usage
|
4342.74 IU/kg/year
STANDARD_DEVIATION 3184.01 • n=5 Participants
|
|
Baseline annualized number of FVIII infusions
|
149.13 Infusions/year
STANDARD_DEVIATION 72.74 • n=5 Participants
|
|
Baseline ABR (treated bleeds)
|
5.61 bleeds/year
STANDARD_DEVIATION 11.29 • n=5 Participants
|
|
Baseline ABR (treated bleeds)
0 bleeds/year
|
5 Participants
n=5 Participants
|
|
Baseline ABR (treated bleeds)
>0 to 4 bleeds/year
|
10 Participants
n=5 Participants
|
|
Baseline ABR (treated bleeds)
>4 to 10 bleeds/year
|
5 Participants
n=5 Participants
|
|
Baseline ABR (treated bleeds)
>10 bleeds/year
|
2 Participants
n=5 Participants
|
|
Baseline ABR (all bleeds)
|
8.20 bleeds/year
STANDARD_DEVIATION 17.86 • n=5 Participants
|
|
Baseline ABR (all bleeds)
0 bleeds/year
|
5 Participants
n=5 Participants
|
|
Baseline ABR (all bleeds)
>0 to 4
|
10 Participants
n=5 Participants
|
|
Baseline ABR (all bleeds)
>4 to 10
|
4 Participants
n=5 Participants
|
|
Baseline ABR (all bleeds)
>10
|
3 Participants
n=5 Participants
|
|
History of previous diseases
Hepatitis B
|
0 Participants
n=5 Participants • n(%)
|
|
History of previous diseases
Hepatitis C
|
3 Participants
n=5 Participants • n(%)
|
|
History of previous diseases
HIV
|
0 Participants
n=5 Participants • n(%)
|
|
History of previous diseases
No History of exposure to Hepatitis B/Hepatitis C/HIV
|
19 Participants
n=5 Participants • n(%)
|
|
Number of target joints
0
|
16 Participants
n=5 Participants
|
|
Number of target joints
1
|
3 Participants
n=5 Participants
|
|
Number of target joints
2
|
2 Participants
n=5 Participants
|
|
Number of target joints
3
|
1 Participants
n=5 Participants
|
|
Adeno-associated virus Type 5 (AAV5) Total Antibody Titers and Positivity
Detected
|
0 Participants
n=5 Participants
|
|
Adeno-associated virus Type 5 (AAV5) Total Antibody Titers and Positivity
Not detected
|
22 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 52Population: Modified intention-to-treat (mITT) population (N=21) - all participants who received BMN 270 infusion in 270-303 and were on adequate prophylactic hemophilia therapy for at least 12 months (adequate prophylaxis was defined as \>= 52 doses of FVIII replacement therapy in 12 months if on FVIII prophylaxis) prior to BMN 270 infusion.
The change from baseline (assuming no treatment for severe hemophilia A) in FVIII activity, as measured by chromogenic substrate assay (CSA), at Week 52 (during Weeks 49 - 52) post-BMN 270 infusion. Each participant's FVIII activity level at Week 52 is defined as the median of the values obtained within the analysis window at Weeks 49-52. The baseline value will be imputed as 1 IU/dL, since there will be no washout of severe hemophilia A participants' usual FVIII prophylaxis (in order to avoid increasing the risk of bleeding) prior to BMN 270 infusion. Post-BMN 270 infusion values for FVIII activity will be excluded from analysis if obtained within 72 hours (or 3 calendar days if time is not available) since the last infusion of exogenous FVIII replacement therapy. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis.
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=21 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in FVIII Activity as Measured by Chromogenic Substrate Assay at Week 52.
|
15.13 IU /dL
Standard Deviation 22.38
|
SECONDARY outcome
Timeframe: Baseline to efficacy evaluation period (EEP)Population: mITT population
The change from baseline (prior to BMN 270 infusion while receiving FVIII prophylaxis) in the annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy in the efficacy evaluation period ("Post-FVIII Prophylaxis period"). The annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy is defined as Sum of FVIII use (IU/kg) during calculation period/Total number of days during the calculation period ×365.25. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit").
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=21 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in Annualized Utilization of Exogenous FVIII Replacement Therapy in EEP
|
-4150.09 IU/kg/yr
Standard Deviation 3208.50
|
SECONDARY outcome
Timeframe: Baseline to efficacy evaluation period (EEP)Population: mITT population
All bleeds comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. All bleeds are any reported bleeding events regardless of the use of FVIII or other treatments. ABR for all bleeds= Number of bleeding episodes for all bleeds during the calculation period / total number of days during the calculation period \* 365.25. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis. EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit").
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=21 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in the Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment (Annualized Bleeding Rate, ABR for All Bleeds) in EEP
|
-6.15 bleeds/year
Standard Deviation 17.24
|
SECONDARY outcome
Timeframe: Baseline to EEPPopulation: mITT population
ABR for treated bleeds=Number of bleeding episodes for treated bleeds during the calculation period/total number of days during the calculation period \* 365.25 Bleeds that were treated with FVIII replacement therapy (recorded as "treatment for bleed") within 72 hours and were not associated with surgery or a procedure were included. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis. EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit").
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=21 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment (ABR for Treated Bleeds) in the EEP.
|
-3.78 bleeds/year
Standard Deviation 10.14
|
SECONDARY outcome
Timeframe: Baseline to Week 52Population: mITT population
The change from baseline(assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at wk52 post-BMN 270 infusion.The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life(HRQoL)questionnaire for adults consisting of 41 items covering 6 domains(Physical Functioning, Role Functioning,Worry,Consequences of Bleeding,Emotional Impact \&Treatment Concerns). The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0(none of the time)to 5 (all of the time).The recall period for the Haemo-Qol-A is one month (4-weeks). The Haemo-QoL-A domain(physical functioning, role functioning, worry, consequences of bleeding, emotional impact, treatment concern) scores range from 0 to 5 and the total score is derived by summing each domain score (range, 0 to 30). Domain and total scores are transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia related quality of life.
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=19 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in Haemo-QoL-A Quality of Life: Total Score at Week 52
|
6.70 score on a scale
Standard Deviation 8.96
|
SECONDARY outcome
Timeframe: Baseline to Week 52Population: mITT population
The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 52 post BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life (HRQoL) questionnaire for adults consisting of 41 items covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns).The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0 (none of the time) to 5 (all of the time).The recall period for the Haemo-Qol-A is one month (4-weeks). The Haemo-Qol-A physical functioning domain score is an average of each item value within a domain.The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The physical functioning domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related physical functioning
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=19 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in Haemo-QoL-A Quality of Life: Physical Functioning Domain Score, at Week 52
|
6.43 score on a scale
Standard Deviation 8.51
|
SECONDARY outcome
Timeframe: Baseline to Week 52Population: mITT population
The change from baseline(assuming no treatment for severe hemophilia A)in Haemo-Qol-A score, at week 52 post-BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life(HRQoL)questionnaire for adults consisting of 41 items covering 6 domains(Physical Functioning,Role Functioning,Worry,Consequences of Bleeding,Emotional Impact and Treatment Concerns). The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0(none of the time) to 5(all of the time). The recall period for the Haemo-Qol-A is one month (4-wks). The Haemo-Qol-A consequences of bleeding domain score is an average of each item value within a domain. The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The consequences of bleeding domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related consequences of bleeding.
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=19 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 52
|
10.68 score on a scale
Standard Deviation 16.33
|
SECONDARY outcome
Timeframe: Baseline to Week 52Population: mITT population
The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 52 post BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life (HRQoL) questionnaire for adults consisting of 41 items covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns).The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0 (none of the time) to 5 (all of the time). The recall period for the Haemo-Qol-A is one month (4-weeks). The Haemo-Qol-A role functioning domain score is an average of each item value within a domain. The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The role functioning domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related role functioning.
Outcome measures
| Measure |
BMN 270 (Valoctocogene roxaparvovec)
n=19 Participants
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
|
|---|---|
|
Change From Baseline in Haemo-QoL-A Quality of Life: Role Functioning Domain Score, at Week 52
|
6.03 Score on a scale
Standard Deviation 10.29
|
Adverse Events
BMN 270 (Valoctocogene Roxaparvovec)
Serious adverse events
| Measure |
BMN 270 (Valoctocogene Roxaparvovec)
n=22 participants at risk
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
|
|---|---|
|
Injury, poisoning and procedural complications
Head injury
|
4.5%
1/22 • Number of events 1 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
Other adverse events
| Measure |
BMN 270 (Valoctocogene Roxaparvovec)
n=22 participants at risk
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
|
|---|---|
|
Investigations
Alanine aminotransferase increased
|
90.9%
20/22 • Number of events 50 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Infections and infestations
COVID-19
|
54.5%
12/22 • Number of events 12 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Skin and subcutaneous tissue disorders
Acne
|
27.3%
6/22 • Number of events 7 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Nervous system disorders
Headache
|
31.8%
7/22 • Number of events 11 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Psychiatric disorders
Insomnia
|
22.7%
5/22 • Number of events 6 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Infections and infestations
Upper respiratory tract infection
|
22.7%
5/22 • Number of events 8 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
18.2%
4/22 • Number of events 4 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Infections and infestations
Influenza
|
18.2%
4/22 • Number of events 4 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
18.2%
4/22 • Number of events 4 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Musculoskeletal and connective tissue disorders
Arthropathy
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Investigations
Aspartate aminotransferase increased
|
18.2%
4/22 • Number of events 9 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
13.6%
3/22 • Number of events 4 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Gastrointestinal disorders
Diarrhoea
|
18.2%
4/22 • Number of events 4 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Gastrointestinal disorders
Dyspepsia
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
General disorders
Fatigue
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
General disorders
Influenza like illness
|
13.6%
3/22 • Number of events 4 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Gastrointestinal disorders
Nausea
|
13.6%
3/22 • Number of events 6 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
18.2%
4/22 • Number of events 4 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
13.6%
3/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Gastrointestinal disorders
Vomiting
|
18.2%
4/22 • Number of events 9 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Psychiatric disorders
Agitation
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Blood and lymphatic system disorders
Anaemia
|
9.1%
2/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Social circumstances
Pregnancy of partner
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
General disorders
Pyrexia
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Infections and infestations
Rash pustular
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Nervous system disorders
Tremor
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Investigations
Gamma-glutamyltransferase increased
|
9.1%
2/22 • Number of events 3 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
|
Infections and infestations
Sinusitis
|
9.1%
2/22 • Number of events 2 • Up to 112 weeks
Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
|
Additional Information
Tara M Robinson, MD, PhD, Senior Medical Director, Late-Stage Clinical Development
BioMarin Pharmaceutical Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60