An Open-Label Study of the Safety and Efficacy of ReFacto AF in Previously Untreated Patients in Usual Care Settings
- Conditions
- Severe hemophilia A (FVIII:C <1%) in male subjects <6 years of age.MedDRA version: 9.1Level: LLTClassification code 10018937Term: Haemophilia A
- Registration Number
- EUCTR2008-008436-93-FR
- Lead Sponsor
- Wyeth Research Division of Wyeth Pharmaceuticals Inc. Clinical Research and Development
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 50
1) Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
2) No prior exposure to factor products or any blood products.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1) Presence of any bleeding disorder in addition to hemophilia A.
2) Treatment with any investigational agent or device within the past 30 days.
3) Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients less than 6 years of age.;Secondary Objective: The secondary objective is to evaluate the efficacy of ReFacto AF.;Primary end point(s): The primary safety outcome is the proportion of patients who develop clinically significant FVIII inhibitors during the course of the study. Clinically significant inhibitors are defined as a central laboratory confirmed positive inhibitor (=0.6 BU using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 4-week interval) and the occurrence of one or more of the<br>following clinical observations: <br>decreased FVIII recovery, need for alternative hemostatic products, increase in number of breakthrough bleeds while on preventive or prophylactic treatment, or more than one report of Less Than Expected Therapeutic Effect (LETE) in the absence of confounding factors.<br>
- Secondary Outcome Measures
Name Time Method