Trial Outcomes & Findings for Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study) (NCT NCT01736475)
NCT ID: NCT01736475
Last Updated: 2021-05-20
Results Overview
Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
159 participants
Primary outcome timeframe
9 months
Results posted on
2021-05-20
Participant Flow
Participants were enrolled (signed informed consent) at 72 sites.
A total of 159 participants provided informed consent and were screened for study participation, of which there were 21 screen failures. 138 participants were assigned to the prophylactic arm or the on-demand treatment regimen.
Participant milestones
| Measure |
Prophylaxis
Twice weekly at a dose of 45 ± 5 IU/kg
|
On-demand
10 to 60 ± 5 IU/kg
|
|---|---|---|
|
Overall Study
STARTED
|
121
|
17
|
|
Overall Study
COMPLETED
|
109
|
17
|
|
Overall Study
NOT COMPLETED
|
12
|
0
|
Reasons for withdrawal
| Measure |
Prophylaxis
Twice weekly at a dose of 45 ± 5 IU/kg
|
On-demand
10 to 60 ± 5 IU/kg
|
|---|---|---|
|
Overall Study
Adverse Event
|
4
|
0
|
|
Overall Study
Withdrawal by Subject
|
2
|
0
|
|
Overall Study
Protocol Violation
|
4
|
0
|
|
Overall Study
Other - Screen failure
|
1
|
0
|
|
Overall Study
Other - Surgical procedure
|
1
|
0
|
Baseline Characteristics
Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)
Baseline characteristics by cohort
| Measure |
Prophylaxis
n=121 Participants
|
On-demand
n=17 Participants
|
Total
n=138 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
29.8 Years
STANDARD_DEVIATION 12.53 • n=5 Participants
|
31.5 Years
STANDARD_DEVIATION 11.05 • n=7 Participants
|
30.0 Years
STANDARD_DEVIATION 12.34 • 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
|
121 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
138 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 9 monthsPopulation: Full Analysis Set
Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy.
Outcome measures
| Measure |
Prophylaxis
n=120 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Annualized Bleeding Rate (ABR)
|
4.3 Bleeds per year
Interval 3.4 to 5.5
|
43.4 Bleeds per year
Interval 25.2 to 74.8
|
SECONDARY outcome
Timeframe: At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm.Population: Full Analysis Set - All bleeding episodes treated with BAX 855 in participants on on-demand and prophylaxis treatment regimens were analyzed as a single group.
Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. EXCELLENT: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. GOOD: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. FAIR: Probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion. Required more than 1 infusion for complete resolution. NONE: No improvement or condition worsens.
Outcome measures
| Measure |
Prophylaxis
n=591 Bleeding episodes
|
On-demand
|
|---|---|---|
|
Rate of Success of BAX 855 for Treatment of Bleeding Episodes
|
0.96 Bleeding episodes
Interval 0.91 to 0.98
|
—
|
SECONDARY outcome
Timeframe: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].Population: Participants from the Full Analysis Set who experienced at least one bleeding episode.
Outcome measures
| Measure |
Prophylaxis
n=65 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes
|
1.37 Infusions
Standard Deviation 0.80
|
1.21 Infusions
Standard Deviation 0.35
|
SECONDARY outcome
Timeframe: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].Population: Study participants from the Full Analysis Set (FAS) who received BAX855 during the study period. Note: one participant was assigned to the prophylactic arm (thus was included in the FAS) and received only ADVATE during the screening period.
Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)\*(12/365.2425)
Outcome measures
| Measure |
Prophylaxis
n=120 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
3 Months
|
11 Participants
|
0 Participants
|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
No bleed
|
45 Participants
|
0 Participants
|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
>6 Months
|
5 Participants
|
0 Participants
|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
6 Months
|
20 Participants
|
0 Participants
|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
5 Months
|
3 Participants
|
0 Participants
|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
4 Months
|
0 Participants
|
0 Participants
|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
2 Months
|
16 Participants
|
0 Participants
|
|
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
≤1 Month
|
20 Participants
|
17 Participants
|
SECONDARY outcome
Timeframe: Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96hPopulation: Full Analysis Set (FAS) - Note: data analyzed by subsets of FAS (1) participants who received BAX855 prophylactic infusion or (2) BAX855 pharmacokinetic (PK) participants. - Subset of participants who received BAX855 prophylactic infusion: N= 120 - Subset of BAX855 pharmacokinetic (PK) participants: N=26
Outcome measures
| Measure |
Prophylaxis
n=5941 Infusions
|
On-demand
|
|---|---|---|
|
Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion
Per Prophylactic Infusion (N= 5941 Infusions)
|
44.51 IU/kg
Standard Deviation 4.556
|
—
|
|
Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion
Per PK Infusion (N= 50 Infusions)
|
45.48 IU/kg
Standard Deviation 2.592
|
—
|
SECONDARY outcome
Timeframe: Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution.Population: Full Analysis Set (FAS) - Note: data analyzed by subsets of FAS (1) participants who received BAX855 for treatment of BEs (2) BAX855 for Maintenance of Hemostasis. - Subset of participants who received BAX855 for treatment of BEs: N= 92 - Subset BAX855 for Maintenance of Hemostasis participants: N=16
Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.
Outcome measures
| Measure |
Prophylaxis
n=592 Bleeds
|
On-demand
|
|---|---|---|
|
Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis
Per Treatment of BE (N= 592 bleeds)
|
37.44 IU/kg
Standard Deviation 28.105
|
—
|
|
Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis
Per BE for Maintenance of Hemostasis (N=34 bleeds)
|
39.29 IU/kg
Standard Deviation 34.206
|
—
|
SECONDARY outcome
Timeframe: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].Population: Safety Analysis Set (SAS) - All participants treated with BAX 855 were analyzed as a single group (ie on-demand and prophylaxis treatment regimens were analyzed as a single group).
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Outcome measures
| Measure |
Prophylaxis
n=137 Participants
|
On-demand
|
|---|---|---|
|
Percentage of Participants With Adverse Events
SAE, Moderate, Unrelated
|
0.7 percent of participants
|
—
|
|
Percentage of Participants With Adverse Events
SAE, Severe, Unrelated
|
2.9 percent of participants
|
—
|
|
Percentage of Participants With Adverse Events
nSAE, Mild, Unrelated
|
40.1 percent of participants
|
—
|
|
Percentage of Participants With Adverse Events
nSAE, Mild, Related
|
3.6 percent of participants
|
—
|
|
Percentage of Participants With Adverse Events
nSAE, Moderate, Unrelated
|
19.0 percent of participants
|
—
|
|
Percentage of Participants With Adverse Events
nSAE, Moderate, Related
|
1.5 percent of participants
|
—
|
|
Percentage of Participants With Adverse Events
nSAE, Unknown Severity, Unrelated
|
0.7 percent of participants
|
—
|
|
Percentage of Participants With Adverse Events
nSAE, Severe, Unrelated
|
1.5 percent of participants
|
—
|
SECONDARY outcome
Timeframe: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].Population: Safety Analysis Set (SAS) - who received BAX855 during the study period. Note: one participant was assigned to the prophylactic arm but did not receive BAX855 (only received ADVATE, during the screening period).
Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary
Outcome measures
| Measure |
Prophylaxis
n=120 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
Inhibitory Antibodies to FVIII (N= 112, 14)
|
0 Participants
|
0 Participants
|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
IgG: Binding Antibodies FVIII (N= 117, 15)
|
0 Participants
|
0 Participants
|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
IgM: Binding Antibodies FVIII (N= 117, 15)
|
0 Participants
|
0 Participants
|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
IgG: Binding Antibodies PEG (N= 117, 15)
|
0 Participants
|
0 Participants
|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
IgM: Binding Antibodies PEG (N= 117, 15)
|
0 Participants
|
0 Participants
|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
IgG: Binding Antibodies PEG-FVIII (N= 117, 15)
|
0 Participants
|
1 Participants
|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
IgM: Binding Antibodies PEG-FVIII (N= 117, 15)
|
0 Participants
|
0 Participants
|
|
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
CHO-Protein Antibodies (N= 117, 15)
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm].Population: Full Analysis Set - Subset of participants with both baseline and study completion HAEMO-SYM scores
The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms.
Outcome measures
| Measure |
Prophylaxis
n=82 Participants
|
On-demand
n=11 Participants
|
|---|---|---|
|
Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study
Bleed Severity Total Score
|
-4.17 Score on a scale
Standard Deviation 17.05
|
-4.24 Score on a scale
Standard Deviation 15.71
|
|
Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study
Pain Severity Total Score
|
-1.22 Score on a scale
Standard Deviation 12.50
|
-0.17 Score on a scale
Standard Deviation 11.88
|
SECONDARY outcome
Timeframe: Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm]Population: Full Analysis Set - Subset of participants with both baseline and study completion SF-36 scores
Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL.
Outcome measures
| Measure |
Prophylaxis
n=97 Participants
|
On-demand
n=12 Participants
|
|---|---|---|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Physical Functioning (N= 97, 12)
|
0.49 Score on a scale
Standard Deviation 5.27
|
-2.46 Score on a scale
Standard Deviation 4.29
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Role-physical (N= 97, 12)
|
1.31 Score on a scale
Standard Deviation 7.36
|
-3.67 Score on a scale
Standard Deviation 9.01
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Bodily Pain (N= 97, 12)
|
2.08 Score on a scale
Standard Deviation 8.19
|
0.60 Score on a scale
Standard Deviation 4.44
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
General Health (N= 96, 12)
|
0.40 Score on a scale
Standard Deviation 6.43
|
-0.28 Score on a scale
Standard Deviation 9.04
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Vitality (N= 96, 12)
|
-0.38 Score on a scale
Standard Deviation 7.43
|
0.26 Score on a scale
Standard Deviation 9.36
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Social Functioning (N= 97, 12)
|
0.90 Score on a scale
Standard Deviation 7.54
|
-3.18 Score on a scale
Standard Deviation 6.35
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Role Emotional (N= 97, 12)
|
-0.20 Score on a scale
Standard Deviation 8.46
|
0.65 Score on a scale
Standard Deviation 7.92
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Mental Health (N= 96, 12)
|
0.09 Score on a scale
Standard Deviation 7.26
|
-3.29 Score on a scale
Standard Deviation 7.95
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Physical Component Score (N= 96, 12)
|
1.36 Score on a scale
Standard Deviation 5.76
|
-1.58 Score on a scale
Standard Deviation 4.97
|
|
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Mental Component Score (N= 96, 12)
|
-0.37 Score on a scale
Standard Deviation 7.38
|
-1.14 Score on a scale
Standard Deviation 5.03
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
Terminal half-life calculated as log\_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)
PK-1: ADVATE
|
10.40 hours
Standard Deviation 2.244
|
—
|
|
Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)
PK-2: BAX 855
|
14.30 hours
Standard Deviation 3.838
|
—
|
|
Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
16.02 hours
Standard Deviation 4.922
|
—
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)
PK-1: ADVATE
|
12.86 hours
Standard Deviation 3.044
|
—
|
|
Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)
PK-2: BAX 855
|
19.56 hours
Standard Deviation 5.315
|
—
|
|
Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
20.65 hours
Standard Deviation 4.821
|
—
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
Clearance in dL/(kg.h) will be calculated as the dose in IU/kg divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)
PK-1: ADVATE
|
0.04551 dL/(kg*hours)
Standard Deviation 0.021725
|
—
|
|
Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)
PK-2: BAX 855
|
0.02760 dL/(kg*hours)
Standard Deviation 0.020288
|
—
|
|
Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
0.02474 dL/(kg*hours)
Standard Deviation 0.008225
|
—
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
Incremental recovery (IR) in (IU/dL)/ (IU/kg) calculated as: IR = (Cmax- (C pre-infusion)) / (Dose/kg), where C =concentration. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)
PK-1: ADVATE
|
2.372 (IU/dL)/(IU/kg)
Standard Deviation 0.5357
|
—
|
|
Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)
PK-2: BAX 855
|
2.493 (IU/dL)/(IU/kg)
Standard Deviation 0.6944
|
—
|
|
Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
2.297 (IU/dL)/(IU/kg)
Standard Deviation 0.6377
|
—
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
Calculated by WinNonlin NCA (Model 201, calculation method: Linear Trapezoidal Linear/Log Interpolation). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)
PK-1: ADVATE
|
1168.0 (IU*hours)/dL
Standard Deviation 425.40
|
—
|
|
Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)
PK-2: BAX 855
|
2073.3 (IU*hours)/dL
Standard Deviation 778.41
|
—
|
|
Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
2008.7 (IU*hours)/dL
Standard Deviation 631.53
|
—
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
The apparent volume of distribution at steady state (Vss) will be calculated as: Vss = Clearance \* Mean Residence Time. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)
PK-1: ADVATE
|
0.5487 dL/kg
Standard Deviation 0.20213
|
—
|
|
Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)
PK-2: BAX 855
|
0.4715 dL/kg
Standard Deviation 0.14602
|
—
|
|
Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
0.4970 dL/kg
Standard Deviation 0.15756
|
—
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)
PK-1: ADVATE
|
108.45 IU/dL
Standard Deviation 26.250
|
—
|
|
Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)
PK-2: BAX 855
|
113.68 IU/dL
Standard Deviation 30.259
|
—
|
|
Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
103.34 IU/dL
Standard Deviation 29.311
|
—
|
SECONDARY outcome
Timeframe: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).Population: Pharmacokinetic full analysis set (PKFAS)
Tmax in hours will be defined as the time to reach Cmax. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Outcome measures
| Measure |
Prophylaxis
n=26 Participants
|
On-demand
|
|---|---|---|
|
Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)
PK-1: ADVATE
|
0.296 hours
Standard Deviation 0.1662
|
—
|
|
Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)
PK-2: BAX 855
|
0.397 hours
Standard Deviation 0.2632
|
—
|
|
Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)
PK-3: BAX 855, After ≥50 Exposure Days (N=22)
|
0.467 hours
Standard Deviation 0.6044
|
—
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/terminationPopulation: Safety Analysis Set
Outcome measures
| Measure |
Prophylaxis
n=119 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Change in Vital Signs From Screening - Temperature
Week 2 (N= 118, 17)
|
0.00 Celsius
Interval -0.2 to 0.2
|
0.00 Celsius
Interval 0.0 to 0.2
|
|
Change in Vital Signs From Screening - Temperature
Week 4 (N= 118, 15)
|
0.00 Celsius
Interval -0.3 to 0.2
|
0.00 Celsius
Interval -0.4 to 0.1
|
|
Change in Vital Signs From Screening - Temperature
Exposure day 10-15 (N= 31, 10)
|
-0.10 Celsius
Interval -0.3 to 0.1
|
0.00 Celsius
Interval -0.3 to 0.3
|
|
Change in Vital Signs From Screening - Temperature
Month 3 (N= 112, 17)
|
0.00 Celsius
Interval -0.2 to 0.2
|
0.00 Celsius
Interval -0.1 to 0.1
|
|
Change in Vital Signs From Screening - Temperature
Completion/Termination (N= 116, 15)
|
0.00 Celsius
Interval -0.2 to 0.2
|
0.00 Celsius
Interval -0.1 to 0.3
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/terminationPopulation: Safety Analysis Set
Outcome measures
| Measure |
Prophylaxis
n=118 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Change in Vital Signs From Screening - Pulse Rate
Week 2 (N= 118, 17)
|
3.0 beats per minute
Interval -3.0 to 9.0
|
2.0 beats per minute
Interval -3.0 to 5.0
|
|
Change in Vital Signs From Screening - Pulse Rate
Week 4 (N= 117, 15)
|
2.0 beats per minute
Interval -4.0 to 10.0
|
2.0 beats per minute
Interval -5.0 to 4.0
|
|
Change in Vital Signs From Screening - Pulse Rate
Exposure day 10-15 (N= 31, 10)
|
3.0 beats per minute
Interval -3.0 to 10.0
|
4.0 beats per minute
Interval 0.0 to 5.0
|
|
Change in Vital Signs From Screening - Pulse Rate
Month 3 (N= 112, 17)
|
2.0 beats per minute
Interval -5.5 to 6.5
|
1.0 beats per minute
Interval -6.0 to 8.0
|
|
Change in Vital Signs From Screening - Pulse Rate
Completion/Termination (N= 116, 15)
|
2.0 beats per minute
Interval -6.0 to 8.5
|
3.0 beats per minute
Interval -5.0 to 10.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/terminationPopulation: Safety Analysis Set
Outcome measures
| Measure |
Prophylaxis
n=118 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Change in Vital Signs From Screening - Respiratory Rate
Week 2 (N= 117, 17)
|
0.0 breaths per minute
Interval -1.0 to 1.0
|
0.0 breaths per minute
Interval -1.0 to 2.0
|
|
Change in Vital Signs From Screening - Respiratory Rate
Week 4 (N= 118, 15)
|
0.0 breaths per minute
Interval -1.0 to 1.0
|
0.0 breaths per minute
Interval -1.0 to 2.0
|
|
Change in Vital Signs From Screening - Respiratory Rate
Exposure day 10-15 (N= 31, 10)
|
0.0 breaths per minute
Interval -1.0 to 2.0
|
0.0 breaths per minute
Interval -1.0 to 0.0
|
|
Change in Vital Signs From Screening - Respiratory Rate
Month 3 (N= 112, 16)
|
0.0 breaths per minute
Interval -2.0 to 2.0
|
0.0 breaths per minute
Interval -2.0 to 1.0
|
|
Change in Vital Signs From Screening - Respiratory Rate
Completion/Termination (N= 115, 15)
|
0.0 breaths per minute
Interval -2.0 to 1.0
|
-1.0 breaths per minute
Interval -2.0 to 2.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/terminationPopulation: Safety Analysis Set
Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP)
Outcome measures
| Measure |
Prophylaxis
n=118 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Vital Signs From Screening - Blood Pressure
SBP-Week 2 (N= 118, 17)
|
0.0 mmHg
Interval -7.0 to 6.0
|
-2.0 mmHg
Interval -9.0 to 7.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
SBP-Week 4 (N= 118, 15)
|
-0.5 mmHg
Interval -9.0 to 7.0
|
-1.0 mmHg
Interval -5.0 to 0.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
SBP-Exposure day 10-15 (N= 31, 10)
|
0.0 mmHg
Interval -5.0 to 4.0
|
3.5 mmHg
Interval 0.0 to 6.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
SBP-Month 3 (N= 112, 17)
|
0.0 mmHg
Interval -7.0 to 7.5
|
-5.0 mmHg
Interval -12.0 to 4.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
SBP-Completion/Termination (N= 116, 15)
|
0.0 mmHg
Interval -10.0 to 7.5
|
0.0 mmHg
Interval -8.0 to 4.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
DBP-Week 2 (N= 118, 17)
|
0.0 mmHg
Interval -8.0 to 5.0
|
-2.0 mmHg
Interval -9.0 to 5.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
DBP-Week 4 (N= 118, 15)
|
-0.5 mmHg
Interval -6.0 to 5.0
|
-6.0 mmHg
Interval -12.0 to 7.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
DBP-Exposure day 10-15 (N= 31, 10)
|
0.0 mmHg
Interval -9.0 to 3.0
|
-2.5 mmHg
Interval -6.0 to 5.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
DBP-Month 3 (N= 112, 17)
|
0.0 mmHg
Interval -7.0 to 7.5
|
-4.0 mmHg
Interval -9.0 to 5.0
|
|
Changes in Vital Signs From Screening - Blood Pressure
DBP-Completion/Termination (N= 116, 15)
|
0.0 mmHg
Interval -6.0 to 5.0
|
-2.0 mmHg
Interval -10.0 to 5.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=115 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Albumin: Week 4 (N= 112, 14)
|
-1.0 g/L
Interval -3.0 to 0.5
|
-1.0 g/L
Interval -2.0 to 0.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Albumin: Week 2 (N= 112, 17)
|
-1.0 g/L
Interval -2.5 to 1.0
|
-1.0 g/L
Interval -2.0 to 0.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Albumin: Month 3 (N= 106, 17)
|
-1.0 g/L
Interval -3.0 to 1.0
|
-1.0 g/L
Interval -2.0 to 1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Albumin: Completion/Termination (N= 112, 14)
|
0.0 g/L
Interval -2.0 to 2.0
|
-1.0 g/L
Interval -2.0 to -1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Protein: Week 2 (N= 115, 17)
|
-1.0 g/L
Interval -4.0 to 1.0
|
-2.0 g/L
Interval -4.0 to 0.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Protein: Week 4 (N= 114, 14)
|
-1.0 g/L
Interval -4.0 to 1.0
|
-2.0 g/L
Interval -5.0 to -1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Protein: Month 3 (N= 109, 17)
|
-1.0 g/L
Interval -3.0 to 2.0
|
-1.0 g/L
Interval -3.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Protein: Completion/Termination (N= 114, 15)
|
-1.0 g/L
Interval -3.0 to 2.0
|
-1.0 g/L
Interval -4.0 to 0.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino)
Outcome measures
| Measure |
Prophylaxis
n=115 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Alk Phos: Week 2 (N= 115, 17)
|
-1.0 Units per Liter
Interval -7.0 to 3.0
|
-5.0 Units per Liter
Interval -7.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Alk Phos: Week 4 (N= 114, 14)
|
-4.0 Units per Liter
Interval -9.0 to 2.0
|
-4.5 Units per Liter
Interval -10.0 to 1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Alk Phos: Month 3 (N= 109, 17)
|
-2.0 Units per Liter
Interval -9.0 to 4.0
|
-5.0 Units per Liter
Interval -11.0 to 4.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Alk Phos: Completion/Termination (N= 114, 15)
|
0.0 Units per Liter
Interval -9.0 to 6.0
|
-5.0 Units per Liter
Interval -9.0 to 1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Ala Amino: Week 2 (N= 112, 17)
|
1.0 Units per Liter
Interval -4.0 to 4.5
|
-2.0 Units per Liter
Interval -6.0 to 5.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Ala Amino: Week 4 (N= 112, 14)
|
0.0 Units per Liter
Interval -5.0 to 4.0
|
-2.5 Units per Liter
Interval -7.0 to 1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Ala Amino: Month 3 (N= 106, 17)
|
1.0 Units per Liter
Interval -6.0 to 5.0
|
1.0 Units per Liter
Interval -4.0 to 12.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Ala Amino: Completion/Termination (N= 112, 14)
|
-1.0 Units per Liter
Interval -7.0 to 4.0
|
2.5 Units per Liter
Interval -3.0 to 5.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Asp Amino: Week 2 (N= 110, 17)
|
0.5 Units per Liter
Interval -2.0 to 5.0
|
-1.0 Units per Liter
Interval -6.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Asp Amino: Week 4 (N= 110, 14)
|
0.0 Units per Liter
Interval -4.0 to 3.0
|
-3.5 Units per Liter
Interval -7.0 to 0.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Asp Amino: Month 3 (N= 103, 17)
|
1.0 Units per Liter
Interval -3.0 to 5.0
|
1.0 Units per Liter
Interval -3.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Asp Amino: Completion/Termination (N= 110, 14)
|
1.0 Units per Liter
Interval -5.0 to 3.0
|
3.5 Units per Liter
Interval -3.0 to 6.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=115 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Chloride: Week 4 (N= 114, 14)
|
1.0 mmol/L
Interval -1.0 to 2.0
|
-1.0 mmol/L
Interval -2.0 to 0.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Bicarbonate: Week 2 (N= 111, 16)
|
0.10 mmol/L
Interval -1.6 to 1.4
|
1.10 mmol/L
Interval -1.5 to 2.6
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Bicarbonate: Week 4 (N= 110, 13)
|
0.40 mmol/L
Interval -0.9 to 2.4
|
0.10 mmol/L
Interval -1.5 to 3.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Bicarbonate: Month 3 (N= 111, 16)
|
0.60 mmol/L
Interval -1.3 to 1.9
|
0.60 mmol/L
Interval -1.3 to 1.75
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Bicarbonate: Completion/Termination (N= 117, 13)
|
1.20 mmol/L
Interval -0.6 to 2.6
|
2.20 mmol/L
Interval 1.0 to 3.9
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Chloride: Week 2 (N= 115, 17)
|
0.0 mmol/L
Interval -1.0 to 2.0
|
-2.0 mmol/L
Interval -3.0 to 1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Chloride: Month 3 (N= 109, 17)
|
0.0 mmol/L
Interval -1.0 to 2.0
|
1.0 mmol/L
Interval -1.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Chloride: Completion/Termination (N= 114, 15)
|
0.0 mmol/L
Interval -1.0 to 2.0
|
-1.0 mmol/L
Interval -2.0 to 1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Glucose: Week 2 (N= 112, 17)
|
-0.10 mmol/L
Interval -0.55 to 0.55
|
-0.30 mmol/L
Interval -0.8 to 0.2
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Glucose: Week 4 (N= 112, 14)
|
0.10 mmol/L
Interval -0.4 to 0.7
|
-0.05 mmol/L
Interval -0.5 to 0.5
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Glucose: Month 3 (N= 106, 17)
|
0.00 mmol/L
Interval -0.5 to 0.5
|
0.10 mmol/L
Interval -0.5 to 0.3
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Glucose: Completion/Termination (N= 115, 14)
|
0.00 mmol/L
Interval -0.5 to 0.6
|
-0.10 mmol/L
Interval -0.5 to 0.6
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Potassium: Week 2 (N= 115, 17)
|
0.00 mmol/L
Interval -0.2 to 0.3
|
0.10 mmol/L
Interval -0.2 to 0.1
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Potassium: Week 4 (N= 114, 14)
|
0.05 mmol/L
Interval -0.2 to 0.3
|
0.10 mmol/L
Interval -0.1 to 0.2
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Potassium: Month 3 (N= 109, 17)
|
0.00 mmol/L
Interval -0.3 to 0.3
|
0.10 mmol/L
Interval -0.1 to 0.4
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Potassium: Completion/Termination (N= 114, 15)
|
0.00 mmol/L
Interval -0.2 to 0.2
|
0.00 mmol/L
Interval -0.1 to 0.4
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Sodium: Week 2 (N= 115, 17)
|
0.0 mmol/L
Interval -1.0 to 1.0
|
-1.0 mmol/L
Interval -2.0 to 1.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Sodium: Week 4 (N= 114, 14)
|
0.0 mmol/L
Interval -1.0 to 1.0
|
-1.5 mmol/L
Interval -3.0 to 0.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Sodium: Month 3 (N= 109, 17)
|
0.0 mmol/L
Interval -2.0 to 1.0
|
0.0 mmol/L
Interval -2.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Sodium: Completion/Termination (N= 114, 15)
|
-1.0 mmol/L
Interval -2.0 to 1.0
|
-1.0 mmol/L
Interval -3.0 to 0.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
BUN: Week 2 (N= 115, 17)
|
0.00 mmol/L
Interval -0.6 to 0.7
|
-0.20 mmol/L
Interval -0.4 to 0.7
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
BUN: Week 4 (N= 113, 14)
|
0.00 mmol/L
Interval -0.7 to 0.7
|
-0.10 mmol/L
Interval -0.3 to 0.5
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
BUN: Month 3 (N= 109, 17)
|
0.30 mmol/L
Interval -0.3 to 0.9
|
-0.40 mmol/L
Interval -0.7 to 0.3
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
BUN: Completion/Termination (N= 114, 15)
|
0.30 mmol/L
Interval -0.7 to 1.1
|
0.00 mmol/L
Interval -1.3 to 1.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=115 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Creatinine: Week 2 (N= 115, 17)
|
0.0 µmol/L
Interval -7.0 to 4.0
|
1.0 µmol/L
Interval -3.0 to 4.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Creatinine: Week 4 (N= 114, 14)
|
0.0 µmol/L
Interval -5.0 to 4.0
|
0.5 µmol/L
Interval -6.0 to 6.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Creatinine: Month 3 (N= 109, 17)
|
0.0 µmol/L
Interval -6.0 to 2.0
|
3.0 µmol/L
Interval -3.0 to 6.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Creatinine: Completion/Termination (N= 114, 15)
|
0.0 µmol/L
Interval -4.0 to 5.0
|
6.0 µmol/L
Interval -4.0 to 9.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Bilirubin: Week 2 (N= 109, 17)
|
0.00 µmol/L
Interval -3.0 to 2.0
|
-2.00 µmol/L
Interval -5.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Bilirubin: Week 4 (N= 109, 14)
|
0.00 µmol/L
Interval -2.0 to 2.0
|
-0.50 µmol/L
Interval -5.0 to 3.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Bilirubin: Month 3 (N= 103, 17)
|
0.00 µmol/L
Interval -3.0 to 2.0
|
1.00 µmol/L
Interval -3.0 to 2.0
|
|
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Bilirubin: Completion/Termination (N= 109, 14)
|
0.00 µmol/L
Interval -3.0 to 2.0
|
-0.50 µmol/L
Interval -2.0 to 3.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=109 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Basophils: Week 2 (N= 108, 17)
|
0.000 giga (10^9) cells per liter (Gi/L)
Interval -0.015 to 0.01
|
0.000 giga (10^9) cells per liter (Gi/L)
Interval -0.01 to 0.02
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Basophils: Week 4 (N= 109, 14)
|
0.000 giga (10^9) cells per liter (Gi/L)
Interval -0.01 to 0.01
|
0.005 giga (10^9) cells per liter (Gi/L)
Interval -0.02 to 0.02
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Basophils: Month 3 (N= 103, 16)
|
0.000 giga (10^9) cells per liter (Gi/L)
Interval -0.02 to 0.02
|
0.000 giga (10^9) cells per liter (Gi/L)
Interval -0.01 to 0.01
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Basophils: Completion/Termination (N= 107, 14)
|
-0.010 giga (10^9) cells per liter (Gi/L)
Interval -0.02 to 0.01
|
0.000 giga (10^9) cells per liter (Gi/L)
Interval -0.02 to 0.02
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Eosinophils: Week 2 (N= 108, 17)
|
0.010 giga (10^9) cells per liter (Gi/L)
Interval -0.02 to 0.05
|
0.030 giga (10^9) cells per liter (Gi/L)
Interval 0.0 to 0.06
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Eosinophils: Week 4 (N= 109, 14)
|
0.020 giga (10^9) cells per liter (Gi/L)
Interval -0.02 to 0.06
|
0.035 giga (10^9) cells per liter (Gi/L)
Interval -0.01 to 0.07
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Eosinophils: Month 3 (N= 103, 16)
|
0.010 giga (10^9) cells per liter (Gi/L)
Interval -0.04 to 0.05
|
0.010 giga (10^9) cells per liter (Gi/L)
Interval -0.005 to 0.05
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Eosinophils: Completion/Termination (N= 107, 14)
|
0.010 giga (10^9) cells per liter (Gi/L)
Interval -0.03 to 0.05
|
0.045 giga (10^9) cells per liter (Gi/L)
Interval 0.01 to 0.08
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Lymphocytes: Week 2 (N= 108, 17)
|
-0.005 giga (10^9) cells per liter (Gi/L)
Interval -0.215 to 0.14
|
0.280 giga (10^9) cells per liter (Gi/L)
Interval -0.07 to 0.64
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Lymphocytes: Week 4 (N= 109, 14)
|
-0.060 giga (10^9) cells per liter (Gi/L)
Interval -0.24 to 0.21
|
0.175 giga (10^9) cells per liter (Gi/L)
Interval 0.03 to 0.35
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Lymphocytes: Month 3 (N= 103, 16)
|
0.030 giga (10^9) cells per liter (Gi/L)
Interval -0.2 to 0.26
|
0.130 giga (10^9) cells per liter (Gi/L)
Interval -0.12 to 0.5
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Lymphocytes: Completion/Termination (N= 107, 14)
|
0.040 giga (10^9) cells per liter (Gi/L)
Interval -0.16 to 0.26
|
0.030 giga (10^9) cells per liter (Gi/L)
Interval -0.07 to 0.46
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Monocytes: Week 2 (N= 108, 17)
|
0.005 giga (10^9) cells per liter (Gi/L)
Interval -0.05 to 0.07
|
0.060 giga (10^9) cells per liter (Gi/L)
Interval -0.05 to 0.13
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Monocytes: Week 4 (N= 109, 14)
|
0.020 giga (10^9) cells per liter (Gi/L)
Interval -0.06 to 0.05
|
-0.010 giga (10^9) cells per liter (Gi/L)
Interval -0.07 to 0.04
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Monocytes: Month 3 (N= 103, 16)
|
0.000 giga (10^9) cells per liter (Gi/L)
Interval -0.06 to 0.05
|
0.020 giga (10^9) cells per liter (Gi/L)
Interval -0.015 to 0.085
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Monocytes: Completion/Termination (N= 107, 14)
|
0.010 giga (10^9) cells per liter (Gi/L)
Interval -0.05 to 0.08
|
0.060 giga (10^9) cells per liter (Gi/L)
Interval -0.04 to 0.08
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Neutrophils: Week 2 (N= 108, 17)
|
-0.165 giga (10^9) cells per liter (Gi/L)
Interval -0.87 to 0.54
|
0.060 giga (10^9) cells per liter (Gi/L)
Interval -0.49 to 0.42
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Neutrophils: Week 4 (N= 109, 14)
|
-0.130 giga (10^9) cells per liter (Gi/L)
Interval -0.68 to 0.45
|
-0.140 giga (10^9) cells per liter (Gi/L)
Interval -0.3 to 0.54
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Neutrophils: Month 3 (N= 103, 16)
|
-0.070 giga (10^9) cells per liter (Gi/L)
Interval -0.86 to 0.49
|
-0.425 giga (10^9) cells per liter (Gi/L)
Interval -0.725 to 0.625
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Neutrophils: Completion/Termination (N= 107, 14)
|
0.050 giga (10^9) cells per liter (Gi/L)
Interval -0.82 to 0.48
|
0.055 giga (10^9) cells per liter (Gi/L)
Interval -0.57 to 1.15
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Platelets: Week 2 (N= 107, 16)
|
-1.0 giga (10^9) cells per liter (Gi/L)
Interval -21.0 to 25.0
|
13.5 giga (10^9) cells per liter (Gi/L)
Interval -6.5 to 34.5
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Platelets: Week 4 (N= 108, 13)
|
2.0 giga (10^9) cells per liter (Gi/L)
Interval -19.5 to 22.5
|
-8.0 giga (10^9) cells per liter (Gi/L)
Interval -18.0 to 7.0
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Platelets: Month 3 (N= 102, 15)
|
1.5 giga (10^9) cells per liter (Gi/L)
Interval -21.0 to 21.0
|
-3.0 giga (10^9) cells per liter (Gi/L)
Interval -24.0 to 17.0
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Platelets: Completion/Termination (N= 105, 13)
|
0.0 giga (10^9) cells per liter (Gi/L)
Interval -20.0 to 20.0
|
-1.0 giga (10^9) cells per liter (Gi/L)
Interval -15.0 to 14.0
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Leukocytes: Week 2 (N= 108, 17)
|
-0.185 giga (10^9) cells per liter (Gi/L)
Interval -0.91 to 0.56
|
0.030 giga (10^9) cells per liter (Gi/L)
Interval -0.55 to 1.08
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Leukocytes: Week 4 (N= 109, 14)
|
-0.180 giga (10^9) cells per liter (Gi/L)
Interval -0.93 to 0.64
|
-0.005 giga (10^9) cells per liter (Gi/L)
Interval -0.67 to 0.75
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Leukocytes: Month 3 (N= 103, 16)
|
-0.040 giga (10^9) cells per liter (Gi/L)
Interval -1.07 to 0.62
|
0.220 giga (10^9) cells per liter (Gi/L)
Interval -0.76 to 1.085
|
|
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Leukocytes: Completion/Termination (N= 107, 14)
|
-0.020 giga (10^9) cells per liter (Gi/L)
Interval -0.9 to 0.68
|
0.250 giga (10^9) cells per liter (Gi/L)
Interval -0.71 to 1.8
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=108 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Hematology Laboratory Assessments From Screening - Hematocrit
Week 2 (N= 107, 17)
|
-0.010 Percentage red blood cells
Interval -0.03 to 0.01
|
0.000 Percentage red blood cells
Interval -0.02 to 0.0
|
|
Changes in Hematology Laboratory Assessments From Screening - Hematocrit
Week 4 (N= 108, 13)
|
-0.010 Percentage red blood cells
Interval -0.02 to 0.005
|
-0.010 Percentage red blood cells
Interval -0.02 to 0.0
|
|
Changes in Hematology Laboratory Assessments From Screening - Hematocrit
Month 3 (N= 102, 16)
|
0.000 Percentage red blood cells
Interval -0.02 to 0.01
|
0.000 Percentage red blood cells
Interval -0.02 to 0.01
|
|
Changes in Hematology Laboratory Assessments From Screening - Hematocrit
Completion/Termination (N= 106, 14)
|
0.000 Percentage red blood cells
Interval -0.01 to 0.02
|
-0.005 Percentage red blood cells
Interval -0.03 to 0.03
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=109 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Hematology Laboratory Assessments From Screening - Hemoglobin
Week 2 (N= 108, 17)
|
-1.0 g/L
Interval -5.0 to 4.0
|
3.0 g/L
Interval -1.0 to 6.0
|
|
Changes in Hematology Laboratory Assessments From Screening - Hemoglobin
Week 4 (N= 109, 14)
|
-1.0 g/L
Interval -5.0 to 5.0
|
0.0 g/L
Interval -2.0 to 7.0
|
|
Changes in Hematology Laboratory Assessments From Screening - Hemoglobin
Month 3 (N= 103, 16)
|
1.0 g/L
Interval -3.0 to 8.0
|
3.5 g/L
Interval -1.5 to 8.5
|
|
Changes in Hematology Laboratory Assessments From Screening - Hemoglobin
Completion/Termination (N= 107, 14)
|
2.0 g/L
Interval -4.0 to 6.0
|
4.5 g/L
Interval -6.0 to 9.0
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=109 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Hematology Laboratory Assessments From Screening - Erythrocytes
Month 3 (N= 103, 16)
|
0.00 TI/L
Interval -0.1 to 0.2
|
0.00 TI/L
Interval -0.1 to 0.2
|
|
Changes in Hematology Laboratory Assessments From Screening - Erythrocytes
Completion/Termination (N= 107, 14)
|
0.00 TI/L
Interval -0.1 to 0.2
|
0.00 TI/L
Interval -0.2 to 0.2
|
|
Changes in Hematology Laboratory Assessments From Screening - Erythrocytes
Week 2 (N= 108, 17)
|
-0.10 TI/L
Interval -0.2 to 0.1
|
0.10 TI/L
Interval -0.2 to 0.1
|
|
Changes in Hematology Laboratory Assessments From Screening - Erythrocytes
Week 4 (N= 109, 14)
|
0.00 TI/L
Interval -0.2 to 0.2
|
0.00 TI/L
Interval -0.2 to 0.1
|
SECONDARY outcome
Timeframe: Screening, week 2, week 4, month 3, study completion/terminationPopulation: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Outcome measures
| Measure |
Prophylaxis
n=115 Participants
|
On-demand
n=17 Participants
|
|---|---|---|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Cholesterol: Month 3 (N= 109, 17)
|
-0.070 mmol/L
Interval -0.33 to 0.36
|
0.040 mmol/L
Interval -0.22 to 0.4
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Cholesterol: Completion/Termination (N= 114, 15)
|
-0.015 mmol/L
Interval -0.34 to 0.32
|
0.080 mmol/L
Interval -0.4 to 0.48
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
HDL: Week 2 (N= 115, 17)
|
0.000 mmol/L
Interval -0.1 to 0.08
|
-0.130 mmol/L
Interval -0.19 to -0.02
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
HDL: Week 4 (N= 114, 14)
|
-0.015 mmol/L
Interval -0.12 to 0.1
|
-0.095 mmol/L
Interval -0.13 to 0.03
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
HDL: Month 3 (N= 109, 17)
|
0.010 mmol/L
Interval -0.11 to 0.1
|
-0.020 mmol/L
Interval -0.13 to 0.05
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
HDL: Completion/Termination (N= 114, 15)
|
-0.020 mmol/L
Interval -0.15 to 0.07
|
0.030 mmol/L
Interval -0.12 to 0.11
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Cholesterol: Week 2 (N= 115, 17)
|
-0.130 mmol/L
Interval -0.39 to 0.2
|
-0.100 mmol/L
Interval -0.77 to 0.26
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Cholesterol: Week 4 (N= 114, 14)
|
-0.050 mmol/L
Interval -0.47 to 0.24
|
-0.190 mmol/L
Interval -0.78 to 0.12
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
LDL: Week 2 (N= 114, 17)
|
-0.070 mmol/L
Interval -0.33 to 0.17
|
-0.090 mmol/L
Interval -0.65 to 0.0
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
LDL: Week 4 (N= 112, 13)
|
-0.080 mmol/L
Interval -0.35 to 0.245
|
-0.320 mmol/L
Interval -0.47 to -0.21
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
LDL: Month 3 (N= 108, 17)
|
-0.030 mmol/L
Interval -0.255 to 0.38
|
0.000 mmol/L
Interval -0.41 to 0.2
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
LDL: Completion/Termination (N= 113, 15)
|
0.050 mmol/L
Interval -0.31 to 0.31
|
-0.050 mmol/L
Interval -0.45 to 0.38
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Triglycerides: Week 2 (N= 115, 17)
|
-0.0600 mmol/L
Interval -0.42 to 0.26
|
0.1700 mmol/L
Interval -0.01 to 0.35
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Triglycerides: Week 4 (N= 114, 14)
|
-0.0800 mmol/L
Interval -0.4 to 0.23
|
0.1500 mmol/L
Interval -0.25 to 0.62
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Triglycerides: Month 3 (N= 109, 17)
|
-0.1600 mmol/L
Interval -0.47 to 0.21
|
0.3600 mmol/L
Interval 0.2 to 0.52
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Triglycerides: Completion/Termination (N= 114, 15)
|
0.0000 mmol/L
Interval -0.38 to 0.23
|
0.1200 mmol/L
Interval -0.04 to 0.21
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
VLDL: Week 2 (N= 114, 17)
|
-0.0150 mmol/L
Interval -0.19 to 0.12
|
0.0800 mmol/L
Interval 0.0 to 0.16
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
VLDL: Week 4 (N= 112, 13)
|
-0.0350 mmol/L
Interval -0.18 to 0.105
|
0.0400 mmol/L
Interval -0.11 to 0.24
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
VLDL: Month 3 (N= 108, 17)
|
-0.0700 mmol/L
Interval -0.225 to 0.095
|
0.1600 mmol/L
Interval 0.09 to 0.24
|
|
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
VLDL: Completion/Termination (N= 113, 15)
|
0.0000 mmol/L
Interval -0.17 to 0.1
|
0.0600 mmol/L
Interval -0.02 to 0.1
|
Adverse Events
All Study Participants
Serious events: 5 serious events
Other events: 26 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Study Participants
n=137 participants at risk
All study participants were analyzed in a single arm/group.
|
|---|---|
|
Infections and infestations
HERPES ZOSTER INFECTION NEUROLOGICAL
|
0.73%
1/137 • Number of events 1 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
|
Injury, poisoning and procedural complications
HUMERUS FRACTURE
|
0.73%
1/137 • Number of events 1 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
|
Musculoskeletal and connective tissue disorders
MUSCLE HAEMORRHAGE
|
0.73%
1/137 • Number of events 1 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
|
Musculoskeletal and connective tissue disorders
OSTEOARTHRITIS
|
0.73%
1/137 • Number of events 1 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
NEUROENDOCRINE CARCINOMA
|
0.73%
1/137 • Number of events 1 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
Other adverse events
| Measure |
All Study Participants
n=137 participants at risk
All study participants were analyzed in a single arm/group.
|
|---|---|
|
Infections and infestations
Nasopharyngitis
|
9.5%
13/137 • Number of events 16 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
|
Infections and infestations
Upper respiratory tract infection
|
6.6%
9/137 • Number of events 10 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
|
Nervous system disorders
Headache
|
5.1%
7/137 • Number of events 13 • From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Baxalta's agreements with PIs may vary per individual PI, but contain common elements. For this study, PIs may be restricted from independently publishing results without prior approval.
- Publication restrictions are in place
Restriction type: OTHER