Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01736475
Recruitment Status : Completed
First Posted : November 29, 2012
Results First Posted : September 7, 2016
Last Update Posted : May 20, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Prevention
Condition Hemophilia A
Interventions Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Biological: PEGylated Recombinant Factor VIII
Enrollment 159
Recruitment Details Participants were enrolled (signed informed consent) at 72 sites.
Pre-assignment Details 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.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description Twice weekly at a dose of 45 ± 5 IU/kg 10 to 60 ± 5 IU/kg
Period Title: Overall Study
Started 121 17
Completed 109 17
Not Completed 12 0
Reason Not Completed
Adverse Event             4             0
Withdrawal by Subject             2             0
Protocol Violation             4             0
Other - Screen failure             1             0
Other - Surgical procedure             1             0
Arm/Group Title Prophylaxis On-demand Total
Hide Arm/Group Description [Not Specified] [Not Specified] Total of all reporting groups
Overall Number of Baseline Participants 121 17 138
Hide Baseline Analysis Population Description
All Study Participants
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 121 participants 17 participants 138 participants
29.8  (12.53) 31.5  (11.05) 30.0  (12.34)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 121 participants 17 participants 138 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
121
 100.0%
17
 100.0%
138
 100.0%
1.Primary Outcome
Title Annualized Bleeding Rate (ABR)
Hide Description 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.
Time Frame 9 months
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 120 17
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Bleeds per year
4.3
(3.4 to 5.5)
43.4
(25.2 to 74.8)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Prophylaxis, On-demand
Comments Ratio Annualized Bleeding Rate (ABR) Prophylaxis/On-demand: Prophylaxis treatment w ill be considered to be successful if the upper limit of the 95% CI for the ratio between treatment regimen does not exceed 0.5 (corresponding to a 50% reduction of the mean ABR compared to the on-demand treatment). H01: μ1 ≥0.5*μ2 Ha1: μ1<0.5*μ2 w here μ1 and μ2 are the mean ABRs in on prophylaxis and on-demand, respectively
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Regression, Negative binomial
Comments [Not Specified]
Method of Estimation Estimation Parameter Ratio of means
Estimated Value 0.10
Confidence Interval (2-Sided) 95%
0.06 to 0.19
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Rate of Success of BAX 855 for Treatment of Bleeding Episodes
Hide Description 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.
Time Frame 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.
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Participants With a Bleeding Episode
Hide Arm/Group Description:
All bleeding episodes treated with BAX 855 in participants on on-demand and prophylaxis treatment regimens.
Overall Number of Participants Analyzed 81
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding episodes
591
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Bleeding episodes
0.96
(0.91 to 0.98)
3.Secondary Outcome
Title Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes
Hide Description [Not Specified]
Time Frame 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].
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Full Analysis Set who experienced at least one bleeding episode.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
Break-through bleeds during prophylaxis
[Not Specified]
Overall Number of Participants Analyzed 65 17
Mean (Standard Deviation)
Unit of Measure: Infusions
1.37  (0.80) 1.21  (0.35)
4.Secondary Outcome
Title Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
Hide Description Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)*(12/365.2425)
Time Frame 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].
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 120 17
Measure Type: Number
Unit of Measure: Participants
No bleed 45 0
>6 Months 5 0
6 Months 20 0
5 Months 3 0
4 Months 0 0
3 Months 11 0
2 Months 16 0
≤1 Month 20 17
5.Secondary Outcome
Title Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion
Hide Description [Not Specified]
Time Frame 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 96h
Hide Outcome Measure Data
Hide Analysis Population Description
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
Arm/Group Title All Study Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 137
Overall Number of Units Analyzed
Type of Units Analyzed: Infusions
5941
Mean (Standard Deviation)
Unit of Measure: IU/kg
Per Prophylactic Infusion (N= 5941 Infusions) 44.51  (4.556)
Per PK Infusion (N= 50 Infusions) 45.48  (2.592)
6.Secondary Outcome
Title Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis
Hide Description Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.
Time Frame 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.
Hide Outcome Measure Data
Hide Analysis Population Description
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
Arm/Group Title All Study Participants
Hide Arm/Group Description:
All Study Participants who received at least one infusion of BAX855.
Overall Number of Participants Analyzed 137
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeds
592
Mean (Standard Deviation)
Unit of Measure: IU/kg
Per Treatment of BE (N= 592 bleeds) 37.44  (28.105)
Per BE for Maintenance of Hemostasis (N=34 bleeds) 39.29  (34.206)
7.Secondary Outcome
Title Percentage of Participants With Adverse Events
Hide Description Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame 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].
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title All Study Participants
Hide Arm/Group Description:
All Study Participants who received at least one infusion of BAX855.
Overall Number of Participants Analyzed 137
Measure Type: Number
Unit of Measure: percent of participants
SAE, Moderate, Unrelated 0.7
SAE, Severe, Unrelated 2.9
nSAE, Mild, Unrelated 40.1
nSAE, Mild, Related 3.6
nSAE, Moderate, Unrelated 19.0
nSAE, Moderate, Related 1.5
nSAE, Unknown Severity, Unrelated 0.7
nSAE, Severe, Unrelated 1.5
8.Secondary Outcome
Title 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
Hide Description 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
Time Frame 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].
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
Twice weekly at a dose of 45 ± 5 IU/kg
10 to 60 ± 5 IU/kg
Overall Number of Participants Analyzed 120 17
Measure Type: Number
Unit of Measure: Participants
Inhibitory Antibodies to FVIII (N= 112, 14) 0 0
IgG: Binding Antibodies FVIII (N= 117, 15) 0 0
IgM: Binding Antibodies FVIII (N= 117, 15) 0 0
IgG: Binding Antibodies PEG (N= 117, 15) 0 0
IgM: Binding Antibodies PEG (N= 117, 15) 0 0
IgG: Binding Antibodies PEG-FVIII (N= 117, 15) 0 1
IgM: Binding Antibodies PEG-FVIII (N= 117, 15) 0 0
CHO-Protein Antibodies (N= 117, 15) 0 0
9.Secondary Outcome
Title Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study
Hide Description 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.
Time Frame 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].
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set - Subset of participants with both baseline and study completion HAEMO-SYM scores
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
Participants with both baseline and study completion HAEMO-SYM scores
Participants with both baseline and study completion HAEMO-SYM scores
Overall Number of Participants Analyzed 82 11
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Bleed Severity Total Score -4.17  (17.05) -4.24  (15.71)
Pain Severity Total Score -1.22  (12.50) -0.17  (11.88)
10.Secondary Outcome
Title Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Hide Description 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.
Time Frame 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]
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set - Subset of participants with both baseline and study completion SF-36 scores
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
Participants with both baseline and study completion SF-36 Scores
Participants with both baseline and study completion SF-36 Scores
Overall Number of Participants Analyzed 97 12
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Physical Functioning (N= 97, 12) 0.49  (5.27) -2.46  (4.29)
Role-physical (N= 97, 12) 1.31  (7.36) -3.67  (9.01)
Bodily Pain (N= 97, 12) 2.08  (8.19) 0.60  (4.44)
General Health (N= 96, 12) 0.40  (6.43) -0.28  (9.04)
Vitality (N= 96, 12) -0.38  (7.43) 0.26  (9.36)
Social Functioning (N= 97, 12) 0.90  (7.54) -3.18  (6.35)
Role Emotional (N= 97, 12) -0.20  (8.46) 0.65  (7.92)
Mental Health (N= 96, 12) 0.09  (7.26) -3.29  (7.95)
Physical Component Score (N= 96, 12) 1.36  (5.76) -1.58  (4.97)
Mental Component Score (N= 96, 12) -0.37  (7.38) -1.14  (5.03)
11.Secondary Outcome
Title Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: hours
PK-1: ADVATE 10.40  (2.244)
PK-2: BAX 855 14.30  (3.838)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 16.02  (4.922)
12.Secondary Outcome
Title Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: hours
PK-1: ADVATE 12.86  (3.044)
PK-2: BAX 855 19.56  (5.315)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 20.65  (4.821)
13.Secondary Outcome
Title Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: dL/(kg*hours)
PK-1: ADVATE 0.04551  (0.021725)
PK-2: BAX 855 0.02760  (0.020288)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 0.02474  (0.008225)
14.Secondary Outcome
Title Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: (IU/dL)/(IU/kg)
PK-1: ADVATE 2.372  (0.5357)
PK-2: BAX 855 2.493  (0.6944)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 2.297  (0.6377)
15.Secondary Outcome
Title Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: (IU*hours)/dL
PK-1: ADVATE 1168.0  (425.40)
PK-2: BAX 855 2073.3  (778.41)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 2008.7  (631.53)
16.Secondary Outcome
Title Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: dL/kg
PK-1: ADVATE 0.5487  (0.20213)
PK-2: BAX 855 0.4715  (0.14602)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 0.4970  (0.15756)
17.Secondary Outcome
Title Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: IU/dL
PK-1: ADVATE 108.45  (26.250)
PK-2: BAX 855 113.68  (30.259)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 103.34  (29.311)
18.Secondary Outcome
Title Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)
Hide Description 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).
Time Frame 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).
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic full analysis set (PKFAS)
Arm/Group Title Pharmacokinetic Analysis Participants
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: hours
PK-1: ADVATE 0.296  (0.1662)
PK-2: BAX 855 0.397  (0.2632)
PK-3: BAX 855, After ≥50 Exposure Days (N=22) 0.467  (0.6044)
19.Secondary Outcome
Title Change in Vital Signs From Screening - Temperature
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 119 17
Median (Inter-Quartile Range)
Unit of Measure: Celsius
Week 2 (N= 118, 17)
0.00
(-0.20 to 0.20)
0.00
(0.00 to 0.20)
Week 4 (N= 118, 15)
0.00
(-0.30 to 0.20)
0.00
(-0.40 to 0.10)
Exposure day 10-15 (N= 31, 10)
-0.10
(-0.30 to 0.10)
0.00
(-0.30 to 0.30)
Month 3 (N= 112, 17)
0.00
(-0.20 to 0.20)
0.00
(-0.10 to 0.10)
Completion/Termination (N= 116, 15)
0.00
(-0.20 to 0.20)
0.00
(-0.10 to 0.30)
20.Secondary Outcome
Title Change in Vital Signs From Screening - Pulse Rate
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 118 17
Median (Inter-Quartile Range)
Unit of Measure: beats per minute
Week 2 (N= 118, 17)
3.0
(-3.0 to 9.0)
2.0
(-3.0 to 5.0)
Week 4 (N= 117, 15)
2.0
(-4.0 to 10.0)
2.0
(-5.0 to 4.0)
Exposure day 10-15 (N= 31, 10)
3.0
(-3.0 to 10.0)
4.0
(0.0 to 5.0)
Month 3 (N= 112, 17)
2.0
(-5.5 to 6.5)
1.0
(-6.0 to 8.0)
Completion/Termination (N= 116, 15)
2.0
(-6.0 to 8.5)
3.0
(-5.0 to 10.0)
21.Secondary Outcome
Title Change in Vital Signs From Screening - Respiratory Rate
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title Prophylaxis On-deamand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 118 17
Median (Inter-Quartile Range)
Unit of Measure: breaths per minute
Week 2 (N= 117, 17)
0.0
(-1.0 to 1.0)
0.0
(-1.0 to 2.0)
Week 4 (N= 118, 15)
0.0
(-1.0 to 1.0)
0.0
(-1.0 to 2.0)
Exposure day 10-15 (N= 31, 10)
0.0
(-1.0 to 2.0)
0.0
(-1.0 to 0.0)
Month 3 (N= 112, 16)
0.0
(-2.0 to 2.0)
0.0
(-2.0 to 1.0)
Completion/Termination (N= 115, 15)
0.0
(-2.0 to 1.0)
-1.0
(-2.0 to 2.0)
22.Secondary Outcome
Title Changes in Vital Signs From Screening - Blood Pressure
Hide Description Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP)
Time Frame Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 118 17
Median (Inter-Quartile Range)
Unit of Measure: mmHg
SBP-Week 2 (N= 118, 17)
0.0
(-7.0 to 6.0)
-2.0
(-9.0 to 7.0)
SBP-Week 4 (N= 118, 15)
-0.5
(-9.0 to 7.0)
-1.0
(-5.0 to 0.0)
SBP-Exposure day 10-15 (N= 31, 10)
0.0
(-5.0 to 4.0)
3.5
(0.0 to 6.0)
SBP-Month 3 (N= 112, 17)
0.0
(-7.0 to 7.5)
-5.0
(-12.0 to 4.0)
SBP-Completion/Termination (N= 116, 15)
0.0
(-10.0 to 7.5)
0.0
(-8.0 to 4.0)
DBP-Week 2 (N= 118, 17)
0.0
(-8.0 to 5.0)
-2.0
(-9.0 to 5.0)
DBP-Week 4 (N= 118, 15)
-0.5
(-6.0 to 5.0)
-6.0
(-12.0 to 7.0)
DBP-Exposure day 10-15 (N= 31, 10)
0.0
(-9.0 to 3.0)
-2.5
(-6.0 to 5.0)
DBP-Month 3 (N= 112, 17)
0.0
(-7.0 to 7.5)
-4.0
(-9.0 to 5.0)
DBP-Completion/Termination (N= 116, 15)
0.0
(-6.0 to 5.0)
-2.0
(-10.0 to 5.0)
23.Secondary Outcome
Title Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 115 17
Median (Inter-Quartile Range)
Unit of Measure: g/L
Albumin: Week 2 (N= 112, 17)
-1.0
(-2.5 to 1.0)
-1.0
(-2.0 to 0.0)
Albumin: Week 4 (N= 112, 14)
-1.0
(-3.0 to 0.5)
-1.0
(-2.0 to 0.0)
Albumin: Month 3 (N= 106, 17)
-1.0
(-3.0 to 1.0)
-1.0
(-2.0 to 1.0)
Albumin: Completion/Termination (N= 112, 14)
0.0
(-2.0 to 2.0)
-1.0
(-2.0 to -1.0)
Protein: Week 2 (N= 115, 17)
-1.0
(-4.0 to 1.0)
-2.0
(-4.0 to 0.0)
Protein: Week 4 (N= 114, 14)
-1.0
(-4.0 to 1.0)
-2.0
(-5.0 to -1.0)
Protein: Month 3 (N= 109, 17)
-1.0
(-3.0 to 2.0)
-1.0
(-3.0 to 2.0)
Protein: Completion/Termination (N= 114, 15)
-1.0
(-3.0 to 2.0)
-1.0
(-4.0 to 0.0)
24.Secondary Outcome
Title Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Hide Description Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino)
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 115 17
Median (Inter-Quartile Range)
Unit of Measure: Units per Liter
Alk Phos: Week 2 (N= 115, 17)
-1.0
(-7.0 to 3.0)
-5.0
(-7.0 to 2.0)
Alk Phos: Week 4 (N= 114, 14)
-4.0
(-9.0 to 2.0)
-4.5
(-10.0 to 1.0)
Alk Phos: Month 3 (N= 109, 17)
-2.0
(-9.0 to 4.0)
-5.0
(-11.0 to 4.0)
Alk Phos: Completion/Termination (N= 114, 15)
0.0
(-9.0 to 6.0)
-5.0
(-9.0 to 1.0)
Ala Amino: Week 2 (N= 112, 17)
1.0
(-4.0 to 4.5)
-2.0
(-6.0 to 5.0)
Ala Amino: Week 4 (N= 112, 14)
0.0
(-5.0 to 4.0)
-2.5
(-7.0 to 1.0)
Ala Amino: Month 3 (N= 106, 17)
1.0
(-6.0 to 5.0)
1.0
(-4.0 to 12.0)
Ala Amino: Completion/Termination (N= 112, 14)
-1.0
(-7.0 to 4.0)
2.5
(-3.0 to 5.0)
Asp Amino: Week 2 (N= 110, 17)
0.5
(-2.0 to 5.0)
-1.0
(-6.0 to 2.0)
Asp Amino: Week 4 (N= 110, 14)
0.0
(-4.0 to 3.0)
-3.5
(-7.0 to 0.0)
Asp Amino: Month 3 (N= 103, 17)
1.0
(-3.0 to 5.0)
1.0
(-3.0 to 2.0)
Asp Amino: Completion/Termination (N= 110, 14)
1.0
(-5.0 to 3.0)
3.5
(-3.0 to 6.0)
25.Secondary Outcome
Title Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 115 17
Median (Inter-Quartile Range)
Unit of Measure: mmol/L
Bicarbonate: Week 2 (N= 111, 16)
0.10
(-1.60 to 1.40)
1.10
(-1.50 to 2.60)
Bicarbonate: Week 4 (N= 110, 13)
0.40
(-0.90 to 2.40)
0.10
(-1.50 to 3.00)
Bicarbonate: Month 3 (N= 111, 16)
0.60
(-1.30 to 1.90)
0.60
(-1.30 to 1.75)
Bicarbonate: Completion/Termination (N= 117, 13)
1.20
(-0.60 to 2.60)
2.20
(1.00 to 3.90)
Chloride: Week 2 (N= 115, 17)
0.0
(-1.0 to 2.0)
-2.0
(-3.0 to 1.0)
Chloride: Week 4 (N= 114, 14)
1.0
(-1.0 to 2.0)
-1.0
(-2.0 to 0.0)
Chloride: Month 3 (N= 109, 17)
0.0
(-1.0 to 2.0)
1.0
(-1.0 to 2.0)
Chloride: Completion/Termination (N= 114, 15)
0.0
(-1.0 to 2.0)
-1.0
(-2.0 to 1.0)
Glucose: Week 2 (N= 112, 17)
-0.10
(-0.55 to 0.55)
-0.30
(-0.80 to 0.20)
Glucose: Week 4 (N= 112, 14)
0.10
(-0.40 to 0.70)
-0.05
(-0.50 to 0.50)
Glucose: Month 3 (N= 106, 17)
0.00
(-0.50 to 0.50)
0.10
(-0.50 to 0.30)
Glucose: Completion/Termination (N= 115, 14)
0.00
(-0.50 to 0.60)
-0.10
(-0.50 to 0.60)
Potassium: Week 2 (N= 115, 17)
0.00
(-0.20 to 0.30)
0.10
(-0.20 to 0.10)
Potassium: Week 4 (N= 114, 14)
0.05
(-0.20 to 0.30)
0.10
(-0.10 to 0.20)
Potassium: Month 3 (N= 109, 17)
0.00
(-0.30 to 0.30)
0.10
(-0.10 to 0.40)
Potassium: Completion/Termination (N= 114, 15)
0.00
(-0.20 to 0.20)
0.00
(-0.10 to 0.40)
Sodium: Week 2 (N= 115, 17)
0.0
(-1.0 to 1.0)
-1.0
(-2.0 to 1.0)
Sodium: Week 4 (N= 114, 14)
0.0
(-1.0 to 1.0)
-1.5
(-3.0 to 0.0)
Sodium: Month 3 (N= 109, 17)
0.0
(-2.0 to 1.0)
0.0
(-2.0 to 2.0)
Sodium: Completion/Termination (N= 114, 15)
-1.0
(-2.0 to 1.0)
-1.0
(-3.0 to 0.0)
BUN: Week 2 (N= 115, 17)
0.00
(-0.60 to 0.70)
-0.20
(-0.40 to 0.70)
BUN: Week 4 (N= 113, 14)
0.00
(-0.70 to 0.70)
-0.10
(-0.30 to 0.50)
BUN: Month 3 (N= 109, 17)
0.30
(-0.30 to 0.90)
-0.40
(-0.70 to 0.30)
BUN: Completion/Termination (N= 114, 15)
0.30
(-0.70 to 1.10)
0.00
(-1.30 to 1.00)
26.Secondary Outcome
Title Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 115 17
Median (Inter-Quartile Range)
Unit of Measure: µmol/L
Creatinine: Week 2 (N= 115, 17)
0.0
(-7.0 to 4.0)
1.0
(-3.0 to 4.0)
Creatinine: Week 4 (N= 114, 14)
0.0
(-5.0 to 4.0)
0.5
(-6.0 to 6.0)
Creatinine: Month 3 (N= 109, 17)
0.0
(-6.0 to 2.0)
3.0
(-3.0 to 6.0)
Creatinine: Completion/Termination (N= 114, 15)
0.0
(-4.0 to 5.0)
6.0
(-4.0 to 9.0)
Bilirubin: Week 2 (N= 109, 17)
0.00
(-3.00 to 2.00)
-2.00
(-5.00 to 2.00)
Bilirubin: Week 4 (N= 109, 14)
0.00
(-2.00 to 2.00)
-0.50
(-5.00 to 3.00)
Bilirubin: Month 3 (N= 103, 17)
0.00
(-3.00 to 2.00)
1.00
(-3.00 to 2.00)
Bilirubin: Completion/Termination (N= 109, 14)
0.00
(-3.00 to 2.00)
-0.50
(-2.00 to 3.00)
27.Secondary Outcome
Title Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 109 17
Median (Inter-Quartile Range)
Unit of Measure: giga (10^9) cells per liter (Gi/L)
Basophils: Week 2 (N= 108, 17)
0.000
(-0.015 to 0.010)
0.000
(-0.010 to 0.020)
Basophils: Week 4 (N= 109, 14)
0.000
(-0.010 to 0.010)
0.005
(-0.020 to 0.020)
Basophils: Month 3 (N= 103, 16)
0.000
(-0.020 to 0.020)
0.000
(-0.010 to 0.010)
Basophils: Completion/Termination (N= 107, 14)
-0.010
(-0.020 to 0.010)
0.000
(-0.020 to 0.020)
Eosinophils: Week 2 (N= 108, 17)
0.010
(-0.020 to 0.050)
0.030
(0.000 to 0.060)
Eosinophils: Week 4 (N= 109, 14)
0.020
(-0.020 to 0.060)
0.035
(-0.010 to 0.070)
Eosinophils: Month 3 (N= 103, 16)
0.010
(-0.040 to 0.050)
0.010
(-0.005 to 0.050)
Eosinophils: Completion/Termination (N= 107, 14)
0.010
(-0.030 to 0.050)
0.045
(0.010 to 0.080)
Lymphocytes: Week 2 (N= 108, 17)
-0.005
(-0.215 to 0.140)
0.280
(-0.070 to 0.640)
Lymphocytes: Week 4 (N= 109, 14)
-0.060
(-0.240 to 0.210)
0.175
(0.030 to 0.350)
Lymphocytes: Month 3 (N= 103, 16)
0.030
(-0.200 to 0.260)
0.130
(-0.120 to 0.500)
Lymphocytes: Completion/Termination (N= 107, 14)
0.040
(-0.160 to 0.260)
0.030
(-0.070 to 0.460)
Monocytes: Week 2 (N= 108, 17)
0.005
(-0.050 to 0.070)
0.060
(-0.050 to 0.130)
Monocytes: Week 4 (N= 109, 14)
0.020
(-0.060 to 0.050)
-0.010
(-0.070 to 0.040)
Monocytes: Month 3 (N= 103, 16)
0.000
(-0.060 to 0.050)
0.020
(-0.015 to 0.085)
Monocytes: Completion/Termination (N= 107, 14)
0.010
(-0.050 to 0.080)
0.060
(-0.040 to 0.080)
Neutrophils: Week 2 (N= 108, 17)
-0.165
(-0.870 to 0.540)
0.060
(-0.490 to 0.420)
Neutrophils: Week 4 (N= 109, 14)
-0.130
(-0.680 to 0.450)
-0.140
(-0.300 to 0.540)
Neutrophils: Month 3 (N= 103, 16)
-0.070
(-0.860 to 0.490)
-0.425
(-0.725 to 0.625)
Neutrophils: Completion/Termination (N= 107, 14)
0.050
(-0.820 to 0.480)
0.055
(-0.570 to 1.150)
Platelets: Week 2 (N= 107, 16)
-1.0
(-21.0 to 25.0)
13.5
(-6.5 to 34.5)
Platelets: Week 4 (N= 108, 13)
2.0
(-19.5 to 22.5)
-8.0
(-18.0 to 7.0)
Platelets: Month 3 (N= 102, 15)
1.5
(-21.0 to 21.0)
-3.0
(-24.0 to 17.0)
Platelets: Completion/Termination (N= 105, 13)
0.0
(-20.0 to 20.0)
-1.0
(-15.0 to 14.0)
Leukocytes: Week 2 (N= 108, 17)
-0.185
(-0.910 to 0.560)
0.030
(-0.550 to 1.080)
Leukocytes: Week 4 (N= 109, 14)
-0.180
(-0.930 to 0.640)
-0.005
(-0.670 to 0.750)
Leukocytes: Month 3 (N= 103, 16)
-0.040
(-1.070 to 0.620)
0.220
(-0.760 to 1.085)
Leukocytes: Completion/Termination (N= 107, 14)
-0.020
(-0.900 to 0.680)
0.250
(-0.710 to 1.800)
28.Secondary Outcome
Title Changes in Hematology Laboratory Assessments From Screening - Hematocrit
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 108 17
Median (Inter-Quartile Range)
Unit of Measure: Percentage red blood cells
Week 2 (N= 107, 17)
-0.010
(-0.030 to 0.010)
0.000
(-0.020 to 0.000)
Week 4 (N= 108, 13)
-0.010
(-0.020 to 0.005)
-0.010
(-0.020 to 0.000)
Month 3 (N= 102, 16)
0.000
(-0.020 to 0.010)
0.000
(-0.020 to 0.010)
Completion/Termination (N= 106, 14)
0.000
(-0.010 to 0.020)
-0.005
(-0.030 to 0.030)
29.Secondary Outcome
Title Changes in Hematology Laboratory Assessments From Screening - Hemoglobin
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 109 17
Median (Inter-Quartile Range)
Unit of Measure: g/L
Week 2 (N= 108, 17)
-1.0
(-5.0 to 4.0)
3.0
(-1.0 to 6.0)
Week 4 (N= 109, 14)
-1.0
(-5.0 to 5.0)
0.0
(-2.0 to 7.0)
Month 3 (N= 103, 16)
1.0
(-3.0 to 8.0)
3.5
(-1.5 to 8.5)
Completion/Termination (N= 107, 14)
2.0
(-4.0 to 6.0)
4.5
(-6.0 to 9.0)
30.Secondary Outcome
Title Changes in Hematology Laboratory Assessments From Screening - Erythrocytes
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 109 17
Median (Inter-Quartile Range)
Unit of Measure: TI/L
Week 2 (N= 108, 17)
-0.10
(-0.20 to 0.10)
0.10
(-0.20 to 0.10)
Week 4 (N= 109, 14)
0.00
(-0.20 to 0.20)
0.00
(-0.20 to 0.10)
Month 3 (N= 103, 16)
0.00
(-0.10 to 0.20)
0.00
(-0.10 to 0.20)
Completion/Termination (N= 107, 14)
0.00
(-0.10 to 0.20)
0.00
(-0.20 to 0.20)
31.Secondary Outcome
Title Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Hide Description [Not Specified]
Time Frame Screening, week 2, week 4, month 3, study completion/termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.
Arm/Group Title Prophylaxis On-demand
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 115 17
Median (Inter-Quartile Range)
Unit of Measure: mmol/L
Cholesterol: Week 2 (N= 115, 17)
-0.130
(-0.390 to 0.200)
-0.100
(-0.770 to 0.260)
Cholesterol: Week 4 (N= 114, 14)
-0.050
(-0.470 to 0.240)
-0.190
(-0.780 to 0.120)
Cholesterol: Month 3 (N= 109, 17)
-0.070
(-0.330 to 0.360)
0.040
(-0.220 to 0.400)
Cholesterol: Completion/Termination (N= 114, 15)
-0.015
(-0.340 to 0.320)
0.080
(-0.400 to 0.480)
HDL: Week 2 (N= 115, 17)
0.000
(-0.100 to 0.080)
-0.130
(-0.190 to -0.020)
HDL: Week 4 (N= 114, 14)
-0.015
(-0.120 to 0.100)
-0.095
(-0.130 to 0.030)
HDL: Month 3 (N= 109, 17)
0.010
(-0.110 to 0.100)
-0.020
(-0.130 to 0.050)
HDL: Completion/Termination (N= 114, 15)
-0.020
(-0.150 to 0.070)
0.030
(-0.120 to 0.110)
LDL: Week 2 (N= 114, 17)
-0.070
(-0.330 to 0.170)
-0.090
(-0.650 to 0.000)
LDL: Week 4 (N= 112, 13)
-0.080
(-0.350 to 0.245)
-0.320
(-0.470 to -0.210)
LDL: Month 3 (N= 108, 17)
-0.030
(-0.255 to 0.380)
0.000
(-0.410 to 0.200)
LDL: Completion/Termination (N= 113, 15)
0.050
(-0.310 to 0.310)
-0.050
(-0.450 to 0.380)
Triglycerides: Week 2 (N= 115, 17)
-0.0600
(-0.4200 to 0.2600)
0.1700
(-0.0100 to 0.3500)
Triglycerides: Week 4 (N= 114, 14)
-0.0800
(-0.4000 to 0.2300)
0.1500
(-0.2500 to 0.6200)
Triglycerides: Month 3 (N= 109, 17)
-0.1600
(-0.4700 to 0.2100)
0.3600
(0.2000 to 0.5200)
Triglycerides: Completion/Termination (N= 114, 15)
0.0000
(-0.3800 to 0.2300)
0.1200
(-0.0400 to 0.2100)
VLDL: Week 2 (N= 114, 17)
-0.0150
(-0.1900 to 0.1200)
0.0800
(0.0000 to 0.1600)
VLDL: Week 4 (N= 112, 13)
-0.0350
(-0.1800 to 0.1050)
0.0400
(-0.1100 to 0.2400)
VLDL: Month 3 (N= 108, 17)
-0.0700
(-0.2250 to 0.0950)
0.1600
(0.0900 to 0.2400)
VLDL: Completion/Termination (N= 113, 15)
0.0000
(-0.1700 to 0.1000)
0.0600
(-0.0200 to 0.1000)
Time Frame 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].
Adverse Event Reporting Description All study participants were analyzed in a single arm/group.
 
Arm/Group Title All Study Participants
Hide Arm/Group Description All study participants were analyzed in a single arm/group.
All-Cause Mortality
All Study Participants
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
All Study Participants
Affected / at Risk (%) # Events
Total   5/137 (3.65%)    
Infections and infestations   
HERPES ZOSTER INFECTION NEUROLOGICAL  1/137 (0.73%)  1
Injury, poisoning and procedural complications   
HUMERUS FRACTURE  1/137 (0.73%)  1
Musculoskeletal and connective tissue disorders   
MUSCLE HAEMORRHAGE  1/137 (0.73%)  1
OSTEOARTHRITIS  1/137 (0.73%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
NEUROENDOCRINE CARCINOMA  1/137 (0.73%)  1
1
Term from vocabulary, MedDRA
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
All Study Participants
Affected / at Risk (%) # Events
Total   26/137 (18.98%)    
Infections and infestations   
Nasopharyngitis  13/137 (9.49%)  16
Upper respiratory tract infection  9/137 (6.57%)  10
Nervous system disorders   
Headache  7/137 (5.11%)  13
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Shire
Phone: +1 866 842 5335
EMail: ClinicalTransparency@shire.com
Layout table for additonal information
Responsible Party: Takeda ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT01736475    
Other Study ID Numbers: 261201
2012-003599-38 ( EudraCT Number )
First Submitted: November 21, 2012
First Posted: November 29, 2012
Results First Submitted: March 4, 2016
Results First Posted: September 7, 2016
Last Update Posted: May 20, 2021