A Phase II/III, multicenter, partially randomized, open label trial investigating safety and efficacy of on-demand and prophylactic treatment with BAY 94-9027 in Severe Hemophilia A
- Conditions
- Hemophilia A10064477
- Registration Number
- NL-OMON45106
- Lead Sponsor
- Bayer
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 9
* Male; 12 to 65 years of age (or Male 18-65 years of age in countries where enrollment of minors is not permitted)
* Subjects with severe hemophilia A (baseline FVIII activity FVIII:C <1%) determined by measurement at the time of screening or from reliable prior documentation (eg, measurement in other clinical trials, result from approved clinical laboratory)
* Previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 150 ED
* Immunocompetent. If HIV positive, CD4+ lymphocyte count >200/mm3
* Current evidence of inhibitor to FVIII with a titer * 0.6 BU/mL, measured by the Nijmegen modified Bethesda assay at the time of screening (central laboratory) (BU: Bethesda unit)
* History of inhibitor to FVIII with a titer * 0.6 BU, or clinical history suggestive of inhibitor requiring modification of treatment. Subjects with a maximum historical titer of 1.0 BU on a single measurement but with at least 3 subsequent successive negative results (< 0.6 BU) thereafter are eligible
* Any other inherited or acquired bleeding disorder in addition to Hemophilia A
* Platelet count < 100,000/mm3
* Creatinine > 2x upper limit of normal
* AST or ALT > 5x upper limit of normal (AST: aspartate aminotransferase; ALT: alanine aminotransferase)
* The subject is currently participating in another investigational drug study, or has participated in a clinical study involving an investigational drug within 30 days of study entry. Subjects who are currently participating in an investigational study in which they are treated with a currently marketed FVIII concentrate are not excluded. Subjects currently treated with BAY 81-8973, and who have received at least 100 ED with the investigational product, may continue treatment with the product up to the start of Visit 1.
* Any subject who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids within the last 3 months. Brief courses of prednisone/methyprednisolone (< 14 days) for treatment of disorders such as synovitis, asthma, etc are allowed at the discretion of the treating physician.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Total number of bleedings as reported by the subjects (efficacy)</p><br>
- Secondary Outcome Measures
Name Time Method <p>- questionnaires (evaluate subject*s assessment)<br /><br>- adverse events (safety and tolerability)<br /><br>- pharmacokinetics and incremental recovery following administration of Bay<br /><br>94-9027<br /><br>- questionnaires for quality of life, work productivity and pain</p><br>