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Trial record 50 of 397 for:    bleeding episodes

Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A

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: NCT00243386
Recruitment Status : Completed
First Posted : October 24, 2005
Results First Posted : November 20, 2012
Last Update Posted : November 20, 2017
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemophilia A
Intervention Drug: Antihemophilic factor, recombinant, manufactured protein-free
Enrollment 82
Recruitment Details Participants were enrolled at 21 European and 9 United States clinical sites beginning January 2006 and completing in June 2010
Pre-assignment Details 82 participants were enrolled and screened. 7 were screen failures, 1 was withdrawn for non-compliance, and 1 requested withdrawal. Therefore, 73 of the 82 enrolled were exposed to investigational product in the first part of the study (on-demand treatment).
Arm/Group Title All Study Participants PK-Driven Prophylaxis Standard Prophylaxis
Hide Arm/Group Description Participants first underwent an open-label pharmacokinetic (PK) evaluation of rAHF-PFM. 48 hours after the initial PK study, a 6 month period of on-demand treatment was conducted (Part 1). After completing the on-demand treatment, participants were randomized to 1 of 2 prophylactic regimens for 12 months ±2 weeks (Part 2). The standard prophylactic regimen was dosed at 20 to 40 IU/kg every 48 ±6 hours, and the PK-driven prophylaxis regimen was dosed at 20 to 80 IU/kg every 72 ±6 hours. The PK-driven prophylaxis regimen was dosed at 20 to 80 IU/kg of rAHF-PFM every 72 ±6 hours The standard prophylactic regimen was dosed at 20 to 40 IU/kg of rAHF-PFM every 48 ±6 hours
Period Title: On-Demand Regimen (Study Part 1)
Started 73 0 [1] 0 [1]
Completed 69 0 0
Not Completed 4 0 0
Reason Not Completed
Withdrawal by Subject             1             0             0
Lost to Follow-up             1             0             0
Screen Failures             2             0             0
[1]
Participants were randomized to PK-Driven or Standard Prophylaxis after Part 1 (On-Demand Regimen)
Period Title: End of Part 1, But Prior to Prophylaxis
Started 69 0 [1] 0 [1]
Completed 66 0 0
Not Completed 3 0 0
Reason Not Completed
Withdrawal by Subject             1             0             0
Lost to Follow-up             1             0             0
Withdrawn for Non-Compliance             1             0             0
[1]
Participants were randomized to PK-Driven or Standard Prophylaxis after Part 1 (On-Demand Regimen)
Period Title: PK-Driven or Standard Prophylaxis
Started 0 [1] 34 32
Completed 0 31 32
Not Completed 0 3 0
Reason Not Completed
Withdrawn for Non-Compliance             0             2             0
Lack of Efficacy             0             1             0
[1]
Participants were randomized to PK-Driven or Standard Prophylaxis after Part 1 (On-Demand Regimen)
Arm/Group Title All Study Participants
Hide Arm/Group Description Participants first underwent an open-label evaluation of rAHF-PFM PK. 48 hours after the initial PK study, a 6 month period of on-demand treatment was conducted (Part 1). After completing the on-demand treatment, participants were randomized to 1 of 2 prophylactic regimens for 12 months ±2 weeks (Part 2). The standard prophylactic regimen was dosed at 20 to 40 IU/kg every 48 ±6 hours, and the PK-driven prophylaxis regimen was dosed at 20 to 80 IU/kg every 72 ±6 hours.
Overall Number of Baseline Participants 73
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 73 participants
26
(7 to 59)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 73 participants
Female
0
   0.0%
Male
73
 100.0%
1.Primary Outcome
Title Mean Transformed Annualized Bleed Rate Estimates From Each of the 1-year Prophylaxis Regimens
Hide Description

Participants were Randomized to Receive 1 of the 2 Following Prophylaxis Regimens (Study Part 2):

  1. Standard prophylaxis (20-40 IU/kg (every 48 ±6 hour), exact regimen determined by investigator)
  2. PK-driven prophylaxis (20-80 IU/kg (every 72 ±6 hour), exact regimen determined by sponsor)

Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X = bleeds/year), X′ = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the t-test.

Time Frame 12 months ±2 weeks
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Hide Analysis Population Description
Per Protocol
Arm/Group Title PK-Driven Prophylaxis Standard Prophylaxis
Hide Arm/Group Description:
PK-driven prophylaxis regimen dosed at 20 to 80 IU/kg every 72 ±6 hours
Standard prophylaxis regimen dosed at 20 to 40 IU/kg every 48 ±6 hours
Overall Number of Participants Analyzed 23 30
Mean (Standard Deviation)
Unit of Measure: (bleeds/year)^(1/2)
1.61  (1.10) 1.46  (0.98)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PK-Driven Prophylaxis, Standard Prophylaxis
Comments

A t-test was used to compare the means of the transformed data. The null-hypothesis tested was H0: X'A(PK-driven prophylaxis) - X'B (standard prophylaxis) = 0 (i.e., no difference for treatment under the 2 prophylactic regimens.

X' = (ABR+0.5)^(1/2)

Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6016
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
2.Primary Outcome
Title Median Annualized Bleed Rate Estimates From Each of the 1 Year Prophylaxis Regimens
Hide Description

Participants were Randomized to Receive 1 of the 2 Following Prophylaxis Regimens (Part 2 of the study):

  1. Standard prophylaxis- infusions every 48 ±6 hours, dosed at 20 to 40 IU/kg.
  2. PK-driven prophylaxis- infusions every 72 ±6 hours dosed at 20 to 80 IU/kg.
Time Frame 12 months ±2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat
Arm/Group Title PK-Driven Prophylaxis Standard Prophylaxis
Hide Arm/Group Description:
PK-driven prophylaxis regimen dosed at 20 to 80 IU/kg every 72 ±6 hours
Standard prophylaxis regimen dosed at 20 to 40 IU/kg every 48 ±6 hours
Overall Number of Participants Analyzed 34 32
Median (Full Range)
Unit of Measure: Bleeds per year
2.01
(0.00 to 17.06)
1.00
(0.00 to 25.87)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PK-Driven Prophylaxis, Standard Prophylaxis
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1467
Comments [Not Specified]
Method Wilcoxon-Rank Sum (Mann-Whitney)
Comments [Not Specified]
3.Secondary Outcome
Title Mean Difference of Transformed Annualized Bleeding Rate Between On-Demand and Standard Prophylaxis Treatment Regimens
Time Frame On-demand 6 months (± 2 weeks); followed by Prophylaxis 12 months (± 2 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat
Arm/Group Title On-Demand Versus Standard Prophylaxis
Hide Arm/Group Description:

On-demand: rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, Gastrointestinal (GI), and intracranial (60-100 IU/kg every 8-12 hours)

Standard Prophylaxis: 20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator

Overall Number of Participants Analyzed 32
Mean (Standard Deviation)
Unit of Measure: (bleeds/year)^(1/2)
5.29  (1.46)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-Demand Versus Standard Prophylaxis
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments 2-sided paired T-tests were done comparing transformed annualized bleeding rates between On-Demand and Prophylaxis regimens.
Method Paired t-Test
Comments [Not Specified]
4.Secondary Outcome
Title Mean Difference of Transformed Annualized Bleeding Rate Between On-Demand and PK-Driven Prophylaxis Treatment Regimens
Time Frame On-demand 6 months (± 2 weeks); followed by Prophylaxis 12 months (± 2 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat
Arm/Group Title On-Demand Versus PK-Driven Prophylaxis
Hide Arm/Group Description:

On-demand: rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours)

PK-driven prophylaxis: 20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor

Overall Number of Participants Analyzed 34
Mean (Standard Deviation)
Unit of Measure: (bleeds/year)^(1/2)
5.00  (1.85)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-Demand Versus PK-Driven Prophylaxis
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments 2-sided paired T-tests were done comparing transformed annualized bleeding rates between On-Demand and Prophylaxis regimens.
Method Paired t-test
Comments [Not Specified]
5.Secondary Outcome
Title Mean Difference of Transformed Annualized Bleeding Rate Between On-Demand and Any Prophylaxis Treatment Regimens
Time Frame On-demand 6 months (± 2 weeks); Prophylaxis 12 months (± 2 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat
Arm/Group Title On-Demand Versus Any Prophylaxis
Hide Arm/Group Description:

On-demand: rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours)

Prophylaxis:

  • Standard prophylaxis: 20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator
  • PK-driven prophylaxis: 20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Overall Number of Participants Analyzed 66
Mean (Standard Deviation)
Unit of Measure: (bleeds/year)^(1/2)
5.14  (1.66)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-Demand Versus Any Prophylaxis
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments 2-sided paired T-tests were done comparing transformed annualized bleeding rates between On-Demand and Prophylaxis regimens.
Method Paired t-test
Comments [Not Specified]
6.Secondary Outcome
Title Total Weight-Adjusted Dose of rAHF-PFM Used Per Year for Each Prophylaxis Arm
Hide Description

Participants were Randomized to Receive 1 of the 2 Following Prophylaxis Regimens (Part 2 of the study):

  1. Standard prophylaxis- infusions every 48 ±6 hours, dosed at 20 to 40 IU/kg.
  2. PK-driven prophylaxis- infusions every 72 ±6 hours dosed at 20 to 80 IU/kg.
Time Frame 12 months ±2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat
Arm/Group Title PK-Driven Prophylaxis Standard Prophylaxis
Hide Arm/Group Description:
PK-driven prophylaxis regimen dosed at 20 to 80 IU/kg every 72 ±6 hours
Standard prophylaxis regimen dosed at 20 to 40 IU/kg every 48 ±6 hours
Overall Number of Participants Analyzed 34 32
Median (Inter-Quartile Range)
Unit of Measure: IU/kg
5197.8
(3268.4 to 8273.5)
5768.2
(4728.0 to 6425.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PK-Driven Prophylaxis, Standard Prophylaxis
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4924
Comments [Not Specified]
Method Wilcoxon-Rank Sum (Mann-Whitney)
Comments [Not Specified]
7.Secondary Outcome
Title Bleeding Episodes Treated With 1 to ≥4 Infusions
Hide Description The number of bleeding episodes treated with 1, 2, 3, or ≥4 infusions of rAHF-PFM to achieve adequate hemostasis
Time Frame Throughout the study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Efficacy Set
Arm/Group Title On-Demand Treatment Standard Prophylaxis Treatment PK-Driven Prophylaxis
Hide Arm/Group Description:
rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours)
20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator
20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Overall Number of Participants Analyzed 62 13 22
Measure Type: Number
Unit of Measure: Bleeding episodes
1 infusion (n = 62, 13, 22) 1168 68 90
2 infusions (n = 51, 6, 9) 277 12 37
3 infusions (n = 27, 2, 4) 128 4 5
4 or more infusions (n = 21, 5, 5) 50 9 7
8.Secondary Outcome
Title Assessment of Hemostasis for Treatment of Bleeding Episodes
Hide Description

Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale):

Excellent: Full pain relief & bleeding cessation within ~8 hrs of 1 infusion. Additional infusions may have been given to maintain hemostasis;

Good: Definite pain relief and/or improvement in bleeding within ~8 hrs after infusion. Possibly requires >1 infusion for complete resolution;

Fair: Probable or slight relief of pain & slight improvement in bleeding within

~8 hrs after infusion. Requires >1 infusion for complete resolution;

None: No improvement or condition worsens

Time Frame On-demand 6 months (± 2 weeks); Prophylaxis 12 months (± 2 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Hemostatic Efficacy Rating Analysis Set (Participants with bleeding episodes that were rated)
Arm/Group Title On-Demand Regimen Standard Prophylaxis PK-Driven Prophylaxis
Hide Arm/Group Description:
rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours)
20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator
20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Overall Number of Participants Analyzed 70 18 25
Measure Type: Number
Unit of Measure: bleeding episodes
Excellent 547 39 33
Good 943 38 75
Fair 167 16 11
None 3 0 20
Unknown 13 0 0
9.Secondary Outcome
Title Total Area Under the Curve (AUC)
Hide Description Total AUC estimated by AUC 0-48h plus an area extrapolated from the log-linear regression model
Time Frame Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Geometric Mean (Standard Deviation)
Unit of Measure: IU*h/dL
1334.45  (454.33) 1061.26  (452.87)
10.Secondary Outcome
Title Area Under the Curve
Hide Description Area under the factor VIII (FVIII) plasma concentration versus time curve (AUC) from 0 to 48 hours estimated using the linear trapezoidal method
Time Frame Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Geometric Mean (Standard Deviation)
Unit of Measure: IU*h/dL
1213.98  (323.96) 966.68  (330.83)
11.Secondary Outcome
Title Maximum Plasma Concentration (C-max)
Hide Description Maximal Factor VIII Concentration After Infusion
Time Frame Within 1 hour post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Geometric Mean (Standard Deviation)
Unit of Measure: IU/dL
91.12  (20.15) 74.47  (11.30)
12.Secondary Outcome
Title Adjusted Incremental Recovery (IR)
Hide Description

Change in factor VIII concentration from pre- to post-infusion at initial and termination study visits.

Adjusted IR defined as:

[Cmax (IU/dL) – pre-infusion FVIII (IU/dL)]/dose (IU/kg)

Time Frame 30 minutes pre-infusion to 48 hours post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Geometric Mean (Standard Deviation)
Unit of Measure: IU/dL per IU/kg
1.81  (0.41) 1.47  (0.27)
13.Secondary Outcome
Title Terminal Half-life
Hide Description Computed from the regression slope in the terminal phase of the model. Terminal half life is the time it takes for the plasma concentration or the amount of drug in the body to be reduced by 50%.
Time Frame Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Mean (Standard Deviation)
Unit of Measure: hours
13.91  (5.07) 14.66  (5.21)
14.Secondary Outcome
Title Weight-Adjusted Clearance
Hide Description Computed as the weight-adjusted dose divided by total AUC
Time Frame Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Mean (Standard Deviation)
Unit of Measure: mL/(kg*h)
3.89  (1.21) 5.17  (1.94)
15.Secondary Outcome
Title Mean Residence Time
Hide Description Computed as total Area Under the Moment Curve (AUMC) divided by the total AUC
Time Frame Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Mean (Standard Deviation)
Unit of Measure: hours
17.71  (7.16) 17.88  (5.67)
16.Secondary Outcome
Title Volume of Distribution at Steady State
Hide Description Computed as weight-adjusted clearance * mean residence time
Time Frame Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Pharmacokinetic Analysis Set
Arm/Group Title ≥14 Years of Age <14 Years of Age
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 65 6
Mean (Standard Deviation)
Unit of Measure: dL/kg
0.65  (0.19) 0.84  (0.19)
17.Secondary Outcome
Title Factor VIII Inhibitor Development
Hide Description Number of treated participants who developed factor VIII inhibitors
Time Frame Throughout study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title All Study Participants
Hide Arm/Group Description:
All participants who were exposed to Investigational Product (IP)
Overall Number of Participants Analyzed 73
Measure Type: Number
Unit of Measure: Participants
0
18.Secondary Outcome
Title Number of Participants With AEs Related to Investigational Product (IP)
Hide Description Number of treated participants with AEs judged to be possibly or probably related to treatment with IP
Time Frame Throughout study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title All Study Participants
Hide Arm/Group Description:
All participants who were exposed to IP
Overall Number of Participants Analyzed 73
Measure Type: Number
Unit of Measure: Participants
4
19.Secondary Outcome
Title Number of Participants Who Reported ≥1 AE Regardless of Relatedness to Investigational Product (IP)
Hide Description Number of treated participants with 1 or more AE regardless of relatedness to IP
Time Frame Throughout study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title All Study Participants
Hide Arm/Group Description:
All participants who were exposed to IP
Overall Number of Participants Analyzed 73
Measure Type: Number
Unit of Measure: Participants
44
20.Secondary Outcome
Title Number of Participants Who Reported ≥1 AE Regardless of Relatedness to IP by Treatment Regimen
Hide Description [Not Specified]
Time Frame Throughout the study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-Demand Standard Prophylaxis PK-Driven Prophylaxis
Hide Arm/Group Description:
rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours)
20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator
20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Overall Number of Participants Analyzed 72 32 34
Measure Type: Number
Unit of Measure: participants
33 15 19
21.Secondary Outcome
Title Number of Participants With SAEs by Preferred MedDRA Term and Treatment Regimen
Hide Description [Not Specified]
Time Frame Throughout the study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-Demand Standard Prophylaxis PK-Driven Prophylaxis SAEs Outside of the 3 Treatment Arms
Hide Arm/Group Description:
[Not Specified]
20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator
20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Participants with SAEs that occurred after exposure to investigational product, but outside of the three treatment arms
Overall Number of Participants Analyzed 72 32 34 1
Measure Type: Number
Unit of Measure: participants
ABDOMINAL PAIN 1 0 0 0
NAUSEA 1 0 0 0
TOOTH ABSCESS 1 0 0 0
JOINT DISLOCATION 1 0 0 0
HAEMOPHILIC ARTHROPATHY 1 0 1 0
SYNOVITIS 1 0 0 0
CALCULUS URINARY 1 0 0 0
HOSPITALIZATION 1 0 0 0
PULPITIS DENTAL 0 1 0 0
SOMNAMBULISM 0 1 0 0
FACTOR VIII INHIBITION (UNCONFIRMED) 0 0 1 0
APPENDICITIS 0 0 1 0
PAIN IN EXTREMITY 0 0 0 1
22.Secondary Outcome
Title AEs With Onset ≤1 Hour Following the End of an Infusion, Regardless of Relatedness
Hide Description [Not Specified]
Time Frame Throughout study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title All Study Participants
Hide Arm/Group Description:
All participants who were exposed to investigational product (IP)
Overall Number of Participants Analyzed 73
Measure Type: Number
Unit of Measure: Events
39
23.Secondary Outcome
Title Number of Participants With Severe SAEs and Severe Non-SAEs by Preferred MedDRA Term and Treatment Regimen
Hide Description This outcome is focused only on SEVERE SAEs and SEVERE non-SAEs
Time Frame Throughout the study period (4 years and 5 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-Demand Standard Prophylaxis PK-Driven Prophylaxis
Hide Arm/Group Description:
rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours
20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator
20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Overall Number of Participants Analyzed 72 32 34
Measure Type: Number
Unit of Measure: participants
TOOTH ABSCESS (SAE) 1 0 0
HAEMOPHILIC ARTHROPATHY (SAE) 0 0 1
HAEMOPHILIC ARTHROPATHY (non-SAE) 1 0 1
ABDOMINAL PAIN (non-SAE) 1 0 0
ARTHRALGIA (non-SAE) 1 0 0
24.Secondary Outcome
Title Baseline Health-related Quality of Life (HRQoL) Scores: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS
Hide Description Physical Functioning (PF); Role Limitation Due to Physical Health (RP); Bodily Pain (BP); General Health (GH); Vitality (VT); Social Functioning (SF); Role Limitation Due to Emotional Problems (RE); Mental Health (MH), Physical Component Score (PCS); Mental Component Score (MCS). Baseline SF-36v1 Scores, where data available. Scores range 0-100, higher scores represent better health. There is no total overall score; scoring is done for subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title Assessed Before Treatment
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 71
Median (Full Range)
Unit of Measure: Scores on a scale
Physical Functioning (PF)
44.56
(17.29 to 57.14)
Role-Physical (RP)
42.10
(27.95 to 56.24)
Bodily Pain (BP)
46.48
(25.07 to 62.75)
General Health (GH)
43.87
(19.52 to 64.00)
Vitality (VT)
51.42
(25.39 to 67.20)
Social Functioning (SF)
46.28
(19.14 to 57.14)
Role-Emotional (RE)
55.34
(23.74 to 55.34)
Mental Health (MH)
50.44
(20.91 to 64.07)
Physical Component Score (PCS)
42.32
(20.10 to 67.67)
Mental Component Score (MCS)
52.65
(22.56 to 68.10)
25.Secondary Outcome
Title Health-related Quality of Life (HRQoL) Scores: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS at the End of Treatment Regimens
Hide Description Physical Functioning (PF); Role Limitation Due to Physical Health (RP); Bodily Pain (BP); General Health (GH); Vitality (VT); Social Functioning (SF); Role Limitation Due to Emotional Problems (RE); Mental Health (MH), Physical Component Score (PCS); Mental Component Score (MCS). Baseline SF-36v1 Scores, where data available. Scores range 0-100, higher scores represent better health. There is no total overall score; scoring is done for subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores.
Time Frame End of on-demand treatment period (6 months) and at study termination (approximately 18 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-Demand Treatment Standard Prophylaxis PK-Driven Prophylaxis Any Prophylaxis Treatment
Hide Arm/Group Description:
rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours
20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator
20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Any Prophylaxis Treatment (either Standard Prophylaxis or PK-Driven Prophylaxis) Standard Prophylaxis: 20-40 IU/kg (every 48 ± 6 hours), exact regimen to be determined by the investigator PK-Driven Prophylaxis: 20-80 IU/kg (every 72 ± 6 hours), exact regimen to be determined by the sponsor
Overall Number of Participants Analyzed 63 31 34 65
Median (Full Range)
Unit of Measure: Scores on a scale
Bodily Pain (BP)
46.5
(29.4 to 62.8)
51.6
(19.9 to 62.8)
51.6
(29.4 to 62.8)
51.6
(19.9 to 62.8)
General Health (GH)
43.9
(19.5 to 64.0)
48.6
(19.5 to 64.0)
46.2
(29.8 to 60.3)
48.6
(19.5 to 64.0)
Mental Component Score (MCS), On-Demand n=62
54.9
(22.8 to 69.9)
56.1
(13.3 to 69.6)
54.5
(11.4 to 62.5)
55.0
(11.4 to 69.6)
Mental Health (MH), On-Demand n=62
51.6
(14.1 to 64.1)
50.4
(20.9 to 64.1)
52.7
(7.3 to 64.1)
50.4
(7.3 to 64.1)
Physical Component Score (PCS), On-Demand n=62
44.0
(16.1 to 61.2)
50.2
(17.6 to 68.8)
47.3
(25.3 to 62.3)
47.8
(17.6 to 68.8)
Physical Functioning (PF), On-Demand n=62
48.8
(17.3 to 57.1)
46.7
(21.5 to 57.1)
46.7
(17.3 to 57.1)
46.7
(17.3 to 57.1)
Role Emotional (RE)
55.3
(23.7 to 55.3)
55.3
(23.7 to 55.3)
55.3
(23.7 to 55.3)
55.3
(23.7 to 55.3)
Role Physical (RP)
49.2
(28.0 to 56.2)
56.2
(28.0 to 56.2)
56.2
(28.0 to 56.2)
56.2
(28.0 to 56.2)
Social Functioning (SF)
46.3
(30.0 to 57.1)
51.7
(24.6 to 57.1)
51.7
(13.7 to 57.1)
51.7
(13.7 to 57.1)
Vitality (VT)
53.8
(32.5 to 70.4)
56.2
(27.8 to 70.4)
56.2
(23.0 to 68.0)
56.2
(23.0 to 70.4)
26.Secondary Outcome
Title HRQoL Scores Change From On-Demand Treatment Regimen Period Through Prophylaxis Period
Hide Description

Differences in health domain scores = (End of on-demand treatment) – (End of prophylaxis regimen). A negative value for the median difference equates to a larger domain score for the prophylaxis regimen

Physical Functioning (PF); Role Limitation Due to Physical Health (RP); Bodily Pain (BP); General Health (GH); Vitality (VT); Social Functioning (SF); Role Limitation Due to Emotional Problems (RE); Mental Health (MH), Physical Component Score (PCS); Mental Component Score (MCS).

Scores range 0-100, higher scores represent better health. There is no total overall score; scoring is done for subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores.

Time Frame End of on-demand treatment period (6 months) and at study termination (approximately 18 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set ≥14 Years and Older
Arm/Group Title On-Demand to Standard Prophylaxis On-Demand to PK-Driven Prophylaxis On-Demand to Any Prophylaxis Treatment
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 27 30 57
Median (Full Range)
Unit of Measure: Scores on a scale
Physical Functioning (PF)
0.00
(-10.48 to 14.68)
-2.10
(-18.88 to 20.97)
-2.10
(-18.88 to 20.97)
Role Physical (RP)
0.00
(-28.29 to 21.21)
0.00
(-28.29 to 28.29)
0.00
(-28.29 to 28.29)
Bodily Pain (BP)
0.00
(-29.55 to 17.55)
-4.29
(-25.27 to 13.28)
0.00
(-29.55 to 17.55)
General Health (GH)
-3.74
(-29.55 to 17.55)
-2.34
(-21.07 to 18.73)
-2.34
(-20.13 to 17.79)
Vitality (VT)
0.00
(-9.47 to 23.67)
0.00
(-16.57 to 30.77)
0.00
(-16.57 to 30.77)
Social Functioning (SF)
0.00
(-21.72 to 16.29)
0.00
(-16.29 to 16.29)
0.00
(-21.72 to 16.29)
Role Emotional (RE)
0.00
(-10.53 to 31.60)
0.00
(-31.60 to 21.07)
0.00
(-31.60 to 31.60)
Mental Health (MH)
0.00
(-13.63 to 15.90)
-1.13
(-40.90 to 34.08)
0.00
(-40.90 to 34.08)
Physical Component Score (PCS)
-2.55
(-20.22 to 8.69)
-3.14
(-16.78 to 19.70)
-2.76
(-20.22 to 19.70)
Mental Component Score (MCS)
1.52
(-8.31 to 21.64)
0.79
(-35.25 to 24.56)
1.30
(-35.25 to 24.56)
27.Secondary Outcome
Title Bodily Pain HRQoL Scores Change From On-Demand Period Through Prophylaxis Period
Hide Description

Change = (End of on-demand treatment) – (End of prophylaxis regimen). A negative value for the median difference equates to a larger domain score for the prophylaxis regimen.

Scores range 0-100, higher scores represent better health. There is no total overall score; scoring is done for subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores.

Time Frame End of on-demand treatment period (6 months) and at study termination (approximately 18 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic analysis set for participants ≥14 years of age
Arm/Group Title ≥14 Years of Age
Hide Arm/Group Description:
After an on-demand treatment period, participants were randomized to 1 of 2 prophylactic regimens for 12 months. The standard prophylactic regimen was dosed at 20 to 40 IU/kg every 48 ±6 hours, and the PK-driven prophylaxis regimen was dosed at 20 to 80 IU/kg every 72 ±6 hours.
Overall Number of Participants Analyzed 57
Median (Full Range)
Unit of Measure: Scores on a scale
0.00
(-29.55 to 17.55)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ≥14 Years of Age
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0007
Comments Due to multiple hypotheses testing results, adjusted alpha values are set to α*=0.005 (0.05/10). Adjustments take into account 10 hypotheses tests between On-Demand and any Prophylaxis. Statistically significant results are considered p-values < α*
Method Wilcoxon signed-rank test
Comments [Not Specified]
28.Secondary Outcome
Title Physical Component Scores (PCS) HRQoL Scores Change From On-Demand Period Through Prophylaxis Period
Hide Description

Change = (End of on-demand treatment) – (End of prophylaxis regimen) A negative value for the median difference equates to a larger domain score for the prophylaxis regimen.

Scores range 0-100, higher scores represent better health. There is no total overall score; scoring is done for subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores.

Time Frame End of on-demand treatment period (6 months) and at study termination (approximately 18 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic analysis set for participants ≥14 years of age
Arm/Group Title ≥14 Years of Age
Hide Arm/Group Description:
After an on-demand treatment period, participants were randomized to 1 of 2 prophylactic regimens for 12 months. The standard prophylactic regimen was dosed at 20 to 40 IU/kg every 48 ±6 hours, and the PK-driven prophylaxis regimen was dosed at 20 to 80 IU/kg every 72 ±6 hours.
Overall Number of Participants Analyzed 57
Median (Full Range)
Unit of Measure: Scores on a scale
-2.76
(-20.22 to 19.70)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ≥14 Years of Age
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments Due to multiple hypotheses testing results, adjusted alpha values are set to α*=0.005 (0.05/10). Adjustments take into account 10 hypotheses tests between On-Demand and any Prophylaxis. Statistically significant results are considered p-values < α*
Method Wilcoxon signed-rank test
Comments [Not Specified]
29.Post-Hoc Outcome
Title Median (IQR) Annualized Bleed Rates
Time Frame On-demand 6 months (± 2 weeks); Prophylaxis 12 months (± 2 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Per-Protocol Efficacy Analysis Set
Arm/Group Title On-Demand Regimen Standard Prophylaxis PK-driven Prophylaxis Any Prophylaxis
Hide Arm/Group Description:
After the initial PK study, a 6 month period of on-demand treatment was conducted (Part 1)
Dosed at 20 to 40 IU/kg every 48 ±6 hours for 12 months (Part 2)
Dosed at 20 to 80 IU/kg every 72 ±6 hours for 12 months (Part 2)
Either Standard or PK-driven Prophylaxis
Overall Number of Participants Analyzed 53 30 23 53
Median (Inter-Quartile Range)
Unit of Measure: Bleeding episodes
43.98
(35.73 to 56.53)
0.99
(0.00 to 2.14)
1.00
(0.00 to 4.08)
1.00
(0.00 to 4.07)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-Demand Regimen, Any Prophylaxis
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Wilcoxon Signed-Rank Test
Comments [Not Specified]
Time Frame Throughout the study period (4 years and 5 months)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title On-Demand Standard Prophylaxis PK-Driven Prophylaxis SAEs Outside of the 3 Treatment Arms
Hide Arm/Group Description On-demand: rAHF-PFM was to be used for the treatment of bleeding episodes according to the severity and type of episode by the protocol-recommended dosing until the episode resolved: superficial (10-20 IU/kg every 12-14 hours), minor joint (20-40 IU/kg every 12-14 hours), deep muscle (30-60 IU/kg every 12-14 hours), major joint or life-threatening (60-100 IU/kg every 8-12 hours), genitourinary, GI, and intracranial (60-100 IU/kg every 8-12 hours) Standard prophylaxis regimen dosed at 20 to 40 IU/kg (every 48 ±6 hours), exact regimen to be determined by the investigator PK-driven prophylaxis regimen dosed at 20 to 80 IU/kg (every 72 ±6 hours) exact regimen to be determined by the sponsor Participants with SAEs that occurred after exposure to investigational product, but outside of the three treatment arms
All-Cause Mortality
On-Demand Standard Prophylaxis PK-Driven Prophylaxis SAEs Outside of the 3 Treatment Arms
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
On-Demand Standard Prophylaxis PK-Driven Prophylaxis SAEs Outside of the 3 Treatment Arms
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/73 (9.59%)      2/32 (6.25%)      3/34 (8.82%)      1/1 (100.00%)    
Blood and lymphatic system disorders         
FACTOR VIII INHIBITION 1 [1]  0/73 (0.00%)  0 0/32 (0.00%)  0 1/34 (2.94%)  1 0/1 (0.00%)  0
Gastrointestinal disorders         
ABDOMINAL PAIN  1/73 (1.37%)  1 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
NAUSEA  1/73 (1.37%)  1 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
Infections and infestations         
TOOTH ABSCESS  1/73 (1.37%)  1 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
PULPITIS DENTAL  0/73 (0.00%)  0 1/32 (3.13%)  1 0/34 (0.00%)  0 0/1 (0.00%)  0
APPENDICITIS  0/73 (0.00%)  0 0/32 (0.00%)  0 1/34 (2.94%)  1 0/1 (0.00%)  0
Injury, poisoning and procedural complications         
JOINT DISLOCATION  1/73 (1.37%)  1 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
Musculoskeletal and connective tissue disorders         
HAEMOPHILIC ARTHROPATHY  1/73 (1.37%)  1 0/32 (0.00%)  0 1/34 (2.94%)  1 0/1 (0.00%)  0
SYNOVITIS  1/73 (1.37%)  1 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
PAIN IN EXTREMITY  0/73 (0.00%)  0 0/32 (0.00%)  0 0/34 (0.00%)  0 1/1 (100.00%)  1
Psychiatric disorders         
SOMNAMBULISM  0/73 (0.00%)  0 1/32 (3.13%)  1 0/34 (0.00%)  0 0/1 (0.00%)  0
Renal and urinary disorders         
CALCULUS URINARY  1/73 (1.37%)  1 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
Surgical and medical procedures         
HOSPITALIZATION  1/73 (1.37%)  1 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
1
Term from vocabulary, MedDRA (Unspecified)
[1]
This event was unconfirmed and therefore did not meet protocol definition for a Factor VIII inhibitor.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
On-Demand Standard Prophylaxis PK-Driven Prophylaxis SAEs Outside of the 3 Treatment Arms
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/73 (24.66%)      4/32 (12.50%)      7/34 (20.59%)      0/1 (0.00%)    
Gastrointestinal disorders         
DIARRHOEA  4/73 (5.48%)  4 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
IRRITABLE BOWEL SYNDROME  0/73 (0.00%)  0 0/32 (0.00%)  0 2/34 (5.88%)  4 0/1 (0.00%)  0
Infections and infestations         
NASOPHARYNGITIS  6/73 (8.22%)  7 0/32 (0.00%)  0 0/34 (0.00%)  0 0/1 (0.00%)  0
UPPER RESPIRATORY TRACT INFECTION  4/73 (5.48%)  4 0/32 (0.00%)  0 2/34 (5.88%)  2 0/1 (0.00%)  0
Musculoskeletal and connective tissue disorders         
ARTHRALGIA  0/73 (0.00%)  0 2/32 (6.25%)  3 0/34 (0.00%)  0 0/1 (0.00%)  0
Nervous system disorders         
HEADACHE  4/73 (5.48%)  6 0/32 (0.00%)  0 3/34 (8.82%)  5 0/1 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
COUGH  0/73 (0.00%)  0 2/32 (6.25%)  2 0/34 (0.00%)  0 0/1 (0.00%)  0
1
Term from vocabulary, MedDRA (Unspecified)
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.
Baxter’s agreements with PIs may vary per requirements of the individual PI, but contain common elements. For this study, PIs are restricted from independently publishing results until the earlier of the primary multicenter publication or ≤2 years after study completion. Baxter requires a review of results communications (e.g., for confidential information) ≥60 days prior to submission or communication. Baxter may request an additional delay of ≤60 days (e.g., intellectual property protection)
Results Point of Contact
Name/Title: Wing-Yen Wong, MD, Global Sr Medical Dir Hemophilia
Organization: Baxter Healthcare Corporation
Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT00243386     History of Changes
Other Study ID Numbers: 060201
First Submitted: October 21, 2005
First Posted: October 24, 2005
Results First Submitted: March 14, 2012
Results First Posted: November 20, 2012
Last Update Posted: November 20, 2017