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Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT00157157
First received: September 9, 2005
Last updated: February 25, 2013
Last verified: February 2013
History of Changes
Results First Received: February 15, 2011  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Hemophilia A
Intervention: Biological: Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Recruitment was conducted in the U.S., Canada, and Europe at 24 study sites.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participants screened for maximum 21 days. Study was not randomized; it was an open-label evaluation. Prior to initial infusion, a minimum washout period of 3 days was required. 11 participants who enrolled did not receive any rAHF-PFM infusions (3 withdrew consent, 6 screen failures, 1 non-compliance with screening, 1 pre-existing low hemoglobin)

Reporting Groups
  Description
Previously Untreated Patients (PUPs) rAHF-PFM was dosed according to a therapeutic regimen which was determined by the investigator (ie: standard regimen [25 to 50 IU/kg body weight, 3 to 4 times per week]; a modified prophylactic regimen [dose and frequency selected by investigator] or on–demand treatment [dose selected by investigator]). The dosing regimen used to treat bleeding episodes (BEs) was at the discretion of the investigator and in accordance with the institution’s standard of care for the type of bleeding episodes diagnosed.

Participant Flow:   Overall Study
    Previously Untreated Patients (PUPs)  
STARTED     55  
COMPLETED     44  
NOT COMPLETED     11  
Withdrawal by Subject                 6  
Physician Decision                 1  
Lost to Follow-up                 1  
Enrolled in another study                 1  
Met exclusion criteria                 1  
Inclusion not met- baseline FVIII value                 1  



  Baseline Characteristics
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Reporting Groups
  Description
PUPs No text entered.

Baseline Measures
    PUPs  
Number of Participants  
[units: participants]
 		  55  
Age, Customized  
[units: participants]
 		 
<6 months     21  
6-12 months     26  
≥13 months     8  
Gender  
[units: participants]
 		 
Female     0  
Male     55  



  Outcome Measures
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1.  Primary:   Factor VIII Inhibitor Development   [ Time Frame: Assessed during study period which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 1

2.  Secondary:   Bleeding Episodes Treated With 1 to ≥4 Infusions   [ Time Frame: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Hide Outcome Measure 2

Measure Type Secondary
Measure Title Bleeding Episodes Treated With 1 to ≥4 Infusions
Measure Description The number of bleeding episodes treated with 1, 2, 3, or ≥4 infusions of rAHF-PFM to achieve adequate hemostasis
Time Frame Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first)  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Participants who received at least 1 infusion of rAHF-PFM for the treatment of bleeding episodes

Reporting Groups
  Description
PUPs No text entered.

Measured Values
    PUPs  
Number of Participants Analyzed  
[units: participants]
 		  44  
Bleeding Episodes Treated With 1 to ≥4 Infusions  
[units: Bleeding episodes]
 		 
1 infusion     356  
2 infusions     107  
3 infusions     35  
4 or more infusions     19  

No statistical analysis provided for Bleeding Episodes Treated With 1 to ≥4 Infusions



3.  Secondary:   Assessment of Hemostasis for Treatment of Bleeding Episodes   [ Time Frame: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 3

4.  Secondary:   Annualized Rate of Bleeding Episodes   [ Time Frame: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 4

5.  Secondary:   Weekly rAHF-PFM Utilization   [ Time Frame: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 5

6.  Secondary:   In Vivo Incremental Recovery   [ Time Frame: 30 minutes pre-infusion to 30 minutes post-infusion ]
  Show Outcome Measure 6

7.  Secondary:   Assessment of Intra-operative Hemostasis   [ Time Frame: Assessed at the time of discharge from recovery room ]
  Show Outcome Measure 7

8.  Secondary:   Assessment of Postoperative Hemostasis   [ Time Frame: Assessed at the time of discharge from hospital or clinic ]
  Show Outcome Measure 8

9.  Secondary:   Assessment of Blood Loss During Surgical Procedures   [ Time Frame: Predicted volumes preoperatively estimated and actual volumes intraoperatively recorded ]
  Show Outcome Measure 9

10.  Secondary:   Adverse Events Deemed Related to Treatment   [ Time Frame: Reported during the study period which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 10

11.  Secondary:   Development of Antibodies to Heterologous Proteins   [ Time Frame: Assessed at baseline, throughout the duration of the study, which was to be at least 75 exposure days or 3 years (whichever came first), and at the termination visit. ]
  Show Outcome Measure 11

12.  Post-Hoc:   Factor VIII Inhibitor Risk Factor: Genetic Risk Factor- Family History of Inhibitors   [ Time Frame: Duration of study which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 12

13.  Post-Hoc:   Factor VIII Inhibitor Risk Factor: Race   [ Time Frame: Duration of study which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 13

14.  Post-Hoc:   Factor VIII Inhibitor Risk Factor: Number of Participants With Intensive Treatment and High Dose (≤20 Exposure Days (EDs))   [ Time Frame: Duration of study which was to be at least 75 exposure days or 3 years (whichever came first) ]
  Show Outcome Measure 14


  Serious Adverse Events
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  Other Adverse Events
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Brigitt Abbuehl, MD, Medical Director, Hematology/Hemophilia
Organization: Baxter Innovations GmbH
e-mail: brigitt_abbuehl@baxter.com


Publications of Results:

Publications automatically indexed to this study:


Responsible Party: Baxter Healthcare Corporation
ClinicalTrials.gov Identifier: NCT00157157     History of Changes
Other Study ID Numbers: 060103
Study First Received: September 9, 2005
Results First Received: February 15, 2011
Last Updated: February 25, 2013
Health Authority: United States: Food and Drug Administration
Germany: Paul-Ehrlich-Institut
Austria: Federal Ministry for Health and Women
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Italy: Ministry of Health
Spain: Spanish Agency of Medicines
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency