Protocol Title: Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C=1%)
- Conditions
- hemophilia AMedDRA version: 20.0Level: LLTClassification code: 10060613Term: Hemophilia A (Factor VIII) Class: 10010331Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2024-512075-12-00
- Lead Sponsor
- Pfizer Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- 75
Males who have been followed on routine Factor VIII prophylaxis therapy in the lead-in study (C0371004) and have = 150 documented exposure days to a Factor VIII protein product, Moderately severe to severe hemophilia A (Factor VIII activity = 1%), Suspension of FVIII prophylaxis therapy post study drug infusion
Anti-AAV6 neutralizing antibodies, History of inhibitor to Factor VIII, Laboratory values at screening visit that are abnormal or outside acceptable study limits, Significant and/or unstable liver disease, biliary disease, significant liver fibrosis, Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit, Active hepatitis B or C, Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with either Cluster of Differentiation 4 positive (CD4+) cell count =200 mm3 or viral load >20 copies/m
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method