Status:
ACTIVE_NOT_RECRUITING
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Lead Sponsor:
Pfizer
Conditions:
Hemophilia A
Eligibility:
MALE
18-64 years
Phase:
PHASE3
Brief Summary
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy...
Eligibility Criteria
Inclusion
- Main inclusion 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
- Main 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
- Conditions associated with increased thromboembolic risk such as inherited or acquired thrombophilia, or a history of thrombotic events
- 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
Exclusion
Key Trial Info
Start Date :
August 18 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 25 2028
Estimated Enrollment :
77 Patients enrolled
Trial Details
Trial ID
NCT04370054
Start Date
August 18 2020
End Date
October 25 2028
Last Update
August 17 2025
Active Locations (36)
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1
NOW Physical Therapy
Mountain View, California, United States, 94040
2
Clinical and Translational Research Unit (CTRU)
Palo Alto, California, United States, 94304
3
Lucile Packard Childrens Hospital
Palo Alto, California, United States, 94304
4
Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
Redwood City, California, United States, 94063