Clinical Trials List
2021-02-15 - 2026-11-26
Phase III
Not yet recruiting3
ICD-10D66
Hereditary factor VIII deficiency
ICD-9286.0
Congenital factor VIII disorder
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%)
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Sponsor
Pfizer
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Trial scale
Multi-Regional Multi-Center
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Update
2026/02/01
Investigators and Locations
Co-Principal Investigator
- 施銘洋 未分科
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- Pei-Chin Lin 未分科
- 許琬宜 未分科
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Condition/Disease
Objectives
Test Drug
Active Ingredient
Dosage Form
Dosage
Endpoints
Inclution Criteria
Males who have been followed on routine Factor VIII prophylaxis therapy during 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
Exclusion Criteria
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 Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 and/or viral load >20 copies/mL
The Estimated Number of Participants
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Taiwan
6 participants
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Global
70 participants
