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BAX 326 (rFIX) Continuation Study

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ClinicalTrials.gov Identifier: NCT01286779
Recruitment Status : Completed
First Posted : January 31, 2011
Results First Posted : September 10, 2018
Last Update Posted : September 10, 2018
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemophilia B
Intervention Biological: BAX 326 (Recombinant factor IX)
Enrollment 117
Recruitment Details Enrollment was conducted at 40 clinical sites in 18 countries. A total of 117 participants were enrolled. Of these, 65 participants transitioned from BAX326 pivotal study, 20 participants transitioned from BAX326 pediatric study and 32 participants were newly recruited.
Pre-assignment Details Of 117 enrolled participants, 115 received treatment with IP. All 85 participants who transitioned from the pivotal/pediatric studies continued to receive IP in this study. Of the 32 newly recruited participants, 30 received treatment with IP. 1 participant did not meet the entry criteria and 1 participant discontinued the study prior treatment.
Arm/Group Title BAX 326
Hide Arm/Group Description Participants treated with BAX 326
Period Title: Overall Study
Started 115
Completed 96
Not Completed 19
Reason Not Completed
Withdrawal by Subject             9
Protocol Violation             5
Physician Decision             2
Participant had scheduled surgery             1
Participant moved to another country             1
Discontinued by sponsor             1
Arm/Group Title BAX 326
Hide Arm/Group Description Participants treated with BAX 326
Overall Number of Baseline Participants 115
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 115 participants
29.6  (16.39)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 115 participants
Female
0
   0.0%
Male
115
 100.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 115 participants
White
99
  86.1%
Black or African American
1
   0.9%
Asian
10
   8.7%
Other
5
   4.3%
1.Primary Outcome
Title Adverse Events Possibly or Probably Related to the Investigational Product
Hide Description Possibly or probably related adverse events that occurred during or after first BAX326 infusion.
Time Frame Assessed (based on patient diary) every 3 months until study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 115
Measure Type: Number
Unit of Measure: Adverse Events
2
2.Secondary Outcome
Title Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Required Until Bleed Resolution
Hide Description Number of Infusions of BAX326 that were required until bleed resolution.
Time Frame Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
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Hide Analysis Population Description
Only bleeding episodes that were exclusively treated with BAX 326 are considered.
Arm/Group Title BAX 326 Standard Prophylaxis Modified Prophylaxis PK Tailored Prophylaxis Overall Prophylaxis On-Demand
Hide Arm/Group Description:
Participants treated with BAX 326
Participants treated with BAX 326 with twice weekly prophylactic infusions of 50 IU/kg
Participants treated with BAX 326 with prophylactic treatment determined by the investigator. The dose could be increased up to 100 IU/kg if indicated.
Participants treated with BAX 326 with PK tailored prophylaxis base on participant's individual PK with maximum dose of 120 IU/kg.
All participants who received BAX 326 as prophylactic regimen (standard prophylaxis, modified prophylaxis and PK-tailored prophylaxis)
All participants who received BAX 326 as on-demand regimen.
Overall Number of Participants Analyzed 115 108 26 3 110 13
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding episodes
1112 542 108 8 658 454
Mean (Standard Deviation)
Unit of Measure: Number of infusions
1.8  (1.65) 2.1  (2.12) 1.9  (1.41) 1.3  (0.46) 2.0  (2.01) 1.5  (0.79)
3.Secondary Outcome
Title Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed
Hide Description

Overall clinical efficacy rating of bleeding episodes was done at resolution of bleed according these rating scale:

Excellent=Full relief of pain and cessation of objective signs of bleeding after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusion would not affect the 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 Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
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Hide Analysis Population Description
Only bleeding episodes that were exclusively treated with BAX 326 are considered.
Arm/Group Title BAX 326 Standard Prophylaxis Modified Prophylaxis PK Tailored Prophylaxis Overall Prophylaxis On-Demand
Hide Arm/Group Description:
Participants treated with BAX 326
Participants treated with BAX 326 with twice weekly prophylactic infusions of 50 IU/kg
Participants treated with BAX 326 with prophylactic treatment determined by the investigator. The dose could be increased up to 100 IU/kg if indicated.
Participants treated with BAX 326 with PK tailored prophylaxis base on participant's individual PK with maximum dose of 120 IU/kg.
All participants who received BAX 326 as prophylactic regimen (standard prophylaxis, modified prophylaxis and PK-tailored prophylaxis)
All participants who received BAX 326 as on-demand regimen.
Overall Number of Participants Analyzed 115 108 26 3 110 13
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes
1112 542 108 8 658 454
Measure Type: Number
Unit of Measure: Number of infusions
Excellent 341 168 51 0 219 122
Good 650 281 40 0 321 329
Fair 115 90 17 6 113 2
None 6 3 0 2 5 1
4.Secondary Outcome
Title Annualized Bleed Rate During Prophylaxis Treatment
Hide Description Annualized bleed rate (ABR) was calculated as (number of bleeding episodes/observed treatment period in days)*365.25
Time Frame For prophylactic treatment the period from first to last prophylactic infusion is considered.
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Hide Analysis Population Description
Only participants with an observation period of at least 3 months with BAX326 on prophylactic treatment were included in the analysis.
Arm/Group Title Standard Prophylaxis Modified Prophylaxis PK Tailored Prophylaxis Overall Prophylaxis
Hide Arm/Group Description:
Participants treated with BAX 326 with twice weekly prophylactic infusions of 50 IU/kg
Participants treated with BAX 326 with prophylactic treatment determined by the investigator. The dose could be increased up to 100 IU/kg if indicated.
Participants treated with BAX 326 with PK tailored prophylaxis base on participant's individual PK with maximum dose of 120 IU/kg.
All participants who received BAX 326 as prophylactic regimen (standard prophylaxis, modified prophylaxis and PK-tailored prophylaxis)
Overall Number of Participants Analyzed 106 22 2 108
Median (Full Range)
Unit of Measure: Bleeds per year
1.3
(0.0 to 78.7)
1.4
(0.0 to 34.6)
1.9
(0.5 to 3.3)
1.3
(0.0 to 52.2)
5.Secondary Outcome
Title Consumption of BAX 326: Number of Infusions Per Month and Per Year
Hide Description The number of infusions consumed per month and per year for the prophylactic and on-demand treatment regimens.
Time Frame Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Standard Prophylaxis Modified Prophylaxis PK Tailored Prophylaxis Overall Prophylaxis On-Demand
Hide Arm/Group Description:
Participants treated with BAX 326 with twice weekly prophylactic infusions of 50 IU/kg
Participants treated with BAX 326 with prophylactic treatment determined by the investigator. The dose could be increased up to 100 IU/kg if indicated.
Participants treated with BAX 326 with PK tailored prophylaxis base on participant's individual PK with maximum dose of 120 IU/kg.
All participants who received BAX 326 as prophylactic regimen (standard prophylaxis, modified prophylaxis and PK-tailored prophylaxis)
All participants who received BAX 326 as on-demand regimen.
Overall Number of Participants Analyzed 108 26 3 110 13
Mean (Standard Deviation)
Unit of Measure: Number of infusions
Number of infusions per month 8.5  (1.25) 10.8  (4.34) 4.0  (0.60) 8.4  (1.38) 3.6  (2.44)
Number of infusions per year 101.8  (15.03) 130.2  (52.13) 48.3  (7.23) 101.1  (16.50) 43.1  (29.28)
6.Secondary Outcome
Title Consumption of BAX 326: Weight Adjusted Consumption Per Month and Per Year
Hide Description The weight adjusted consumption of BAX 326 per month and per year for the prophylactic and on-demand treatment regimens.
Time Frame Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Standard Prophylaxis Modified Prophylaxis PK Tailored Prophylaxis Overall Prophylaxis On-Demand
Hide Arm/Group Description:
Participants treated with BAX 326 with twice weekly prophylactic infusions of 50 IU/kg
Participants treated with BAX 326 with prophylactic treatment determined by the investigator. The dose could be increased up to 100 IU/kg if indicated.
Participants treated with BAX 326 with PK tailored prophylaxis base on participant's individual PK with maximum dose of 120 IU/kg.
All participants who received BAX 326 as prophylactic regimen (standard prophylaxis, modified prophylaxis and PK-tailored prophylaxis)
All participants who received BAX 326 as on-demand regimen.
Overall Number of Participants Analyzed 108 26 3 110 13
Mean (Standard Deviation)
Unit of Measure: IU/kg
Weight adjusted BAX 326 consumption per month 462.3  (102.05) 684.4  (337.70) 250.9  (41.37) 464.2  (111.46) 199.8  (124.18)
Weight adjusted BAX 326 consumption per year 5547.8  (1224.65) 8212.4  (4052.36) 3010.3  (496.44) 5570.7  (1337.53) 2397.4  (1490.22)
7.Secondary Outcome
Title Consumption of BAX326: Weight Adjusted Consumption Per Bleeding Episode
Hide Description The weight adjusted consumption of BAX 326 per bleeding episode for the prophylactic and on-demand treatment regimens. Only infusions required until the resolution of bleed are considered.
Time Frame Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Standard Prophylaxis Modified Prophylaxis PK Tailored Prophylaxis Overall Prophylaxis On-Demand
Hide Arm/Group Description:
Participants treated with BAX 326 with twice weekly prophylactic infusions of 50 IU/kg
Participants treated with BAX 326 with prophylactic treatment determined by the investigator. The dose could be increased up to 100 IU/kg if indicated.
Participants treated with BAX 326 with PK tailored prophylaxis base on participant's individual PK with maximum dose of 120 IU/kg.
All participants who received BAX 326 as prophylactic regimen (standard prophylaxis, modified prophylaxis and PK-tailored prophylaxis)
All participants who received BAX 326 as on-demand regimen.
Overall Number of Participants Analyzed 108 26 3 110 13
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding episodes
542 109 8 659 453
Mean (Standard Deviation)
Unit of Measure: IU/kg
124.2  (140.70) 114.8  (99.41) 67.4  (34.39) 122.0  (134.02) 82.6  (48.21)
8.Secondary Outcome
Title Development of Inhibitory and Total Binding Antibodies to Factor IX
Hide Description

Testing for inhibitory and total binding antibodies to Factor IX (FIX). Development during study means negative at screening and positive at any subsequent visit.

Treatment emergent means more than 2-dilution increase as compared to the pre-study titer at screening.

Time Frame Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.
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[Not Specified]
Arm/Group Title BAX 326
Hide Arm/Group Description:
Participants treated with BAX 326
Overall Number of Participants Analyzed 115
Measure Type: Count of Participants
Unit of Measure: Participants
Inhibitory antibodies to FIX-develop. during study
0
   0.0%
Inhibitory antibodies to FIX-treatment emergent
0
   0.0%
Total bind. antibodies to FIX-develop.during study
0
   0.0%
Total binding antibodies to FIX-treatment emergent
0
   0.0%
9.Secondary Outcome
Title Development of Antibodies to Chinese Hamster Ovary Proteins (CHO Proteins) and rFurin
Hide Description

Testing for antibodies to CHO proteins and rFurin. Development during study means negative at screening and positive at any subsequent visit.

Treatment emergent means more than 2-dilution increase as compared to the pre-study titer at screening.

Time Frame Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.
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[Not Specified]
Arm/Group Title BAX 326
Hide Arm/Group Description:
Participants treated with BAX 326
Overall Number of Participants Analyzed 115
Measure Type: Count of Participants
Unit of Measure: Participants
Antibodies to CHO - developed during study
0
   0.0%
Antibodies to CHO - treatment emergent
0
   0.0%
Antibodies to rFurin - developed during study
4
   3.5%
Antibodies to rFurin - treatment emergent
4
   3.5%
10.Secondary Outcome
Title Occurrence of Severe Allergic Reactions and Thrombotic Events
Hide Description The occurrence of severe allergic reactions and thrombotic events was assessed.
Time Frame Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
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[Not Specified]
Arm/Group Title BAX 326
Hide Arm/Group Description:
Participants treated with BAX 326
Overall Number of Participants Analyzed 115
Measure Type: Count of Participants
Unit of Measure: Participants
Severe allergic reactions
0
   0.0%
Thrombotic events
0
   0.0%
11.Secondary Outcome
Title Clinical Significant Changes in Routine Laboratory Parameters and Vital Signs
Hide Description

Hematology panel consists of complete blood count (hemoglobin, hematocrit, erythrocytes, leukocytes) with differential (ie, basophils, eosinophils, lymphocytes, monocytes, neutrophils), mean corpuscular volume, mean corpuscular hemoglobin concentration and platelet count.

Clinical chemistry panel consists of sodium, potassium, chloride, bicarbonate, total protein, albumin, alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, alkaline phosphatase, blood urea nitrogen, creatinine and glucose.

Vital signs include body temperature, respiratory rate, pulse rate, supine systolic and diastolic blood pressure. CS=clinically significant, NCS=not clinically significant. Change from Screening to End of Study is reported.

Time Frame Measurements at screening and at study completion/termination are included in the analysis.
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[Not Specified]
Arm/Group Title BAX 326
Hide Arm/Group Description:
Participants treated with BAX 326
Overall Number of Participants Analyzed 115
Measure Type: Count of Participants
Unit of Measure: Participants
Hematology: Change from normal to abnormal CS
2
   1.7%
Hematology:Change from abnormal NCS to abnormal CS
1
   0.9%
Chemistry: Change from normal to abnormal, CS
1
   0.9%
Chemistry: Change from abnormal NCS to abnormal CS
3
   2.6%
Change in vital signs
0
   0.0%
12.Secondary Outcome
Title Pharmacokinetics: Incremental Recovery (IR) Over Time
Hide Description PK infusion with investigational product was administered after a wash out period of at least 5 days. Incremental recovery is calculated as IR30min = (C30min [IU/dL] - Cpre-infusion [IU/dL]) / dose per kg body weight [IU/kg] where C30min and Cpre-infusion relate to the unadjusted concentration values.
Time Frame IR over time was measured as Baseline and at Completion/Termination visit within 30 minutes pre-infusion and at 30 (± 5) minutes post-infusion.
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Hide Analysis Population Description
Analysis was done on all participants who received investigational product. All cases with a dosage higher than 120 IU/kg were excluded from the analysis.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 115
Mean (Standard Deviation)
Unit of Measure: (IU/dL):(IU/kg)
Baseline Number Analyzed 110 participants
0.85  (0.207)
End of Study Number Analyzed 108 participants
0.85  (0.286)
Change from baseline to end of study Number Analyzed 104 participants
-0.005  (0.259)
13.Secondary Outcome
Title Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-∞)
Hide Description After a wash out period of at least 5 days PK infusion with investigational product was administered. AUC 0-∞ is defined as AUC 0-t + Ct/lambda z, where t is the time of last quantifiable concentration, Ct is the last quantifiable concentration.
Time Frame PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
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The pharmacokinetic analysis set comprises all participants who underwent an abbreviated PK study.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: IU*hr/dL
1335.56  (299.83)
14.Secondary Outcome
Title Pharmacokinetics: Elimination Phase Half-life (T1/2)
Hide Description PK infusion with investigational product was administered after a wash out period of at least 5 days. Elimination phase half-life is calculated as T1/2=log e (2) / lambda z where the elimination rate constant (lambda z) will be obtained by log e - linear fitting using least squares deviation to at least the last 3 quantifiable concentrations above pre-infusion level.
Time Frame PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
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Hide Analysis Population Description
The pharmacokinetic analysis set comprises all participants who underwent an abbreviated PK study.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: hours
28.52  (4.12)
15.Secondary Outcome
Title Pharmacokinetics: Mean Residence Time (MRT)
Hide Description PK infusion with investigational product was administered after a wash out period of at least 5 days. Mean residence time is calculated as total area under the moment curve divided by the total area under the curve. MRT=(AUMC0-∞[h2*IU/dL])/(AUC0-∞[h*IU/dL]) - TI/2 where AUMC0-∞ is determined in a similar manner as AUC0-∞ and TI represents infusion duration in hours.
Time Frame PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
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Hide Analysis Population Description
The pharmacokinetic analysis set comprises all participants who underwent an abbreviated PK study.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: hours
29.97  (2.72)
16.Secondary Outcome
Title Pharmacokinetics: Systemic Clearance (CL)
Hide Description PK infusion with investigational product was administered after a wash out period of at least 5 days. Systemic clearance is balculated as the dose in IU/kg divided by the total AUC. CL= Dose[IU/kg] / AUC0-∞[h*IU/dL]
Time Frame PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
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Hide Analysis Population Description
The pharmacokinetic analysis set comprises all participants who underwent an abbreviated PK study.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: dL/kg/hours
0.06  (0.014)
17.Secondary Outcome
Title Pharmacokinetics: Volume of Distribution at Steady State (Vss)
Hide Description PK infusion with investigational product was administered after a wash out period of at least 5 days. Apparent steady state volume of distribution is calculated as Vss = CL * MRT CL=Systemic Clearance and MRT=Mean residence time
Time Frame PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
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Hide Analysis Population Description
The pharmacokinetic analysis set comprises all participants who underwent an abbreviated PK study.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: dL/kg
1.78  (0.42)
18.Secondary Outcome
Title Pharmacokinetics: Incremental Recovery (IR)
Hide Description PK infusion with investigational product was administered after a wash out period of at least 5 days. Incremental recovery is calculated as IR30min = (C30min [IU/dL] - Cpre-infusion [IU/dL]) / dose per kg body weight [IU/kg] where C30min and Cpre-infusion relate to the unadjusted concentration values.
Time Frame PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours.
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Hide Analysis Population Description
The pharmacokinetic analysis set comprises all participants who underwent an abbreviated PK study.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: (IU/dL):(IU/kg)
0.85  (0.196)
19.Secondary Outcome
Title Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire SF-36
Hide Description The Short Form (36) Health Survey (SF-36) is a 36-item validated, generic HR QoL instrument suitable for participants of 17 years of age or older. The SF-36 consists of eight scaled scores (vitality, physical functioning, bodily pain, general health, mental health, physical role functioning, emotional role functioning, social role functioning) which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability. The mental health component summary score ranged from 19.5 to 64.2 with higher scores indicating less disability. The physical health component summary scores ranged from 18.6 to 59.6 with higher scores indicating less disability.
Time Frame Baseline at exposure day 1 and at study completion/termination.
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Hide Analysis Population Description
Only newly recruited participants are included as baseline values were not reported for transitioning participants. Only subjects how received prophylaxis treatment are included.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 24
Mean (Standard Deviation)
Unit of Measure: Score on a scale
SF-36 Bodily Pain 6.7  (12.66)
SF-36 General Health 3.2  (9.32)
SF-36 Mental Health 0.7  (14.72)
SF-36 Mental Health Component Score 0.8  (12.08)
SF-36 Physical Functioning 4.2  (10.46)
SF-36 Physical Health Component Score 5.7  (8.43)
SF-36 Role-Emotional 2.3  (11.57)
SF-36 Role-Physical 4.5  (10.28)
SF-36 Social Functioning 4.5  (11.67)
SF-36 Vitality 2.0  (8.87)
20.Secondary Outcome
Title Changes in Health Related Quality of Life Using the Peds QL
Hide Description

The Pediatric Quality of Life Inventory (Peds QL) is a generic health related quality of life instrument designed specifically for a pediatric population and captures following domains: physical functioning, emotional functioning, social functioning and school functioning.

The Peds-QL total score consist of all 23 items of all domains. Score range from 0-100 and higher scores indicate better quality of life (collected scores ranged from 44.6 to 98.9).

The Peds-QL Physical Health Summary score consists of 8 items from the physical functioning domain. Score range from 0-100 and higher scores indicate better quality of life (collected scores ranged from 40.6 to 100.0) The Psychosocial Health Summary score consists of 15 items from the emotional, social and school functioning domains. Score range from 0 to 100 and higher scores indicate better quality of life (collected scores ranged from 46.7 to 100.0).

Time Frame Baseline at exposure day 1 and at study completion/termination.
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Hide Analysis Population Description
Only newly recruited participants are included as baseline values were not reported for transitioning participants. Only subjects how received prophylaxis treatment are included.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Peds-QL Physical Health Summary Score -2.3  (18.47)
Peds-QL Psychosocial Health Summary Score 3.8  (5.99)
Peds-QL Total Score 1.6  (10.14)
21.Secondary Outcome
Title Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire Haemo-QoL and Haem-A-QoL
Hide Description

The Hemophilia Quality of Life Questionnaire (Haemo-QoL) and the Hemophilia Quality of Life Questionnaire for Adults (Haem-A-Qol) instruments have been developed and used in hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: physical health, sports/leisure, school/work, dealing with hemophilia, and outlook for the future.

Haemo-QoL is used for participants aged 8 to 16 years and total scores range from 0 to 100 with higher scores indicating low quality of life (collected scores ranged from 0.0 to 44.3) Haem-A-QoL is used for participants aged 17 years and older and total scores range from 0 to 100 with higher scores indicating low quality of life (collected scores ranged from 4.9 to 76.8).

Time Frame Baseline at exposure day 1 and at study completion/termination.
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Hide Analysis Population Description
Only newly recruited participants are included as baseline values were not reported for transitioning participants. Only subjects how received prophylaxis treatment are included.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Haem-A-QoL Total Score Number Analyzed 16 participants
-3.0  (9.45)
Haemo-QoL Total Score Number Analyzed 1 participants
-0.7 [1]   (NA)
[1]
only 1 participant analyzed
22.Secondary Outcome
Title Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire EQ-5D and Pain Score.
Hide Description

The EQ-5D captures overall HR QoL (phyiscal, mental and social functioning). A health utility score can be calculated from this measure, adult and proxy versions available.

EQ-5D Visual Analog Scale (EQ-5D VAS):Respondents specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints (scale range from 0 to 100). Score 0 corresponds to the worst health you can imagine and score 100 corresponds to the best health you can imagine (collected scores ranged from 10-100).

EQ-5D Total Index is based on general population valuation surveys. Responses to 5 questions are converted to an Index value and score range from 0 to 1, with higher scores indicating better quality of life. Total Index was derived on US population (collected scores ranged from 0.4-1).

General pain assessment (Pain score) is done through a visual analog scale (VAS), scores ranging from 0 to 100 with higher scores indicating more pain (collected scores ranged 0-87).

Time Frame Baseline at exposure day 1 and at study completion/termination.
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Hide Analysis Population Description
Only newly recruited participants are included as baseline values were not reported for transitioning participants. Only subjects how received prophylaxis treatment are included.
Arm/Group Title BAX 326
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Participants treated with BAX 326
Overall Number of Participants Analyzed 26
Mean (Standard Deviation)
Unit of Measure: Score on a scale
EQ-5D Total Index Number Analyzed 26 participants
0.0  (0.13)
EQ-5D VAS Number Analyzed 25 participants
5.1  (21.75)
Pain Score Number Analyzed 24 participants
-8.0  (36.62)
Time Frame Throughout the entire study period from screening to completion/termination. Overall 6 years and 2 months. For each participant the duration of study depended on when the participant has accumulated a total of 100 exposure days during the course of the pivotal/pediatric studies and this study.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title BAX 326
Hide Arm/Group Description Participants treated with BAX 326
All-Cause Mortality
BAX 326
Affected / at Risk (%)
Total   0/115 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
BAX 326
Affected / at Risk (%) # Events
Total   9/115 (7.83%)    
Gastrointestinal disorders   
Abdominal pain * 1  1/115 (0.87%)  1
Duodenal ulcer hemorrhage * 1  1/115 (0.87%)  1
Infections and infestations   
Corneal abscess * 1  1/115 (0.87%)  1
Injury, poisoning and procedural complications   
Brain contusion * 1  1/115 (0.87%)  1
Extradural hematoma * 1  1/115 (0.87%)  1
Head injury * 1  1/115 (0.87%)  1
Scroctal haematoma * 1  1/115 (0.87%)  1
Nervous system disorders   
Seizure * 1  1/115 (0.87%)  1
Transient ischaemic attack * 1  1/115 (0.87%)  1
Renal and urinary disorders   
Haematuria * 1  1/115 (0.87%)  3
Renal colic * 1  2/115 (1.74%)  3
Reproductive system and breast disorders   
Testicular appendage torsion * 1  1/115 (0.87%)  1
1
Term from vocabulary, MedDRA
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
BAX 326
Affected / at Risk (%) # Events
Total   63/115 (54.78%)    
General disorders   
Pyrexia * 1  14/115 (12.17%)  23
Infections and infestations   
Bronchitis * 1  6/115 (5.22%)  8
Influenza * 1  8/115 (6.96%)  8
Nasopharyngitis * 1  25/115 (21.74%)  55
Pharyngitis * 1  6/115 (5.22%)  8
Rhinitis * 1  8/115 (6.96%)  15
Upper respiratory tract infection * 1  11/115 (9.57%)  17
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  15/115 (13.04%)  48
Nervous system disorders   
Headache * 1  8/115 (6.96%)  20
Respiratory, thoracic and mediastinal disorders   
Cough * 1  11/115 (9.57%)  15
Oropharyngeal pain * 1  6/115 (5.22%)  6
Vascular disorders   
Hypertension * 1  6/115 (5.22%)  6
1
Term from vocabulary, MedDRA
*
Indicates events were collected by non-systematic assessment
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.
Agreements may vary with individual PIs, but contain common elements. For this study, PIs are restricted from independently publishing results until the earlier of the primary multicenter publication or up to 2 years after study completion. The sponsor requires a review of results communication (e .g. for confidential information) ≥ 60 days prior to submission and may request an additional delay up to 6 months (e .g. for intellectual property protection). Prior authorization may be required.
Results Point of Contact
Name/Title: Study Director
Organization: Baxalta, now part of Shire
Phone: +1 866 842 5335
Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT01286779     History of Changes
Other Study ID Numbers: 251001
2010-022726-33 ( EudraCT Number )
First Submitted: January 26, 2011
First Posted: January 31, 2011
Results First Submitted: June 21, 2018
Results First Posted: September 10, 2018
Last Update Posted: September 10, 2018