A Study to Evaluate the Efficacy and Safety of Factor VIII Gene Therapy With PF-07055480 in Adult Males With Moderately Severe to Severe Hemophilia A
- Conditions
- hemophilia AMedDRA version: 20.0Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2019-004451-37-DE
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 63
• 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 63
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
• 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/mL
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