Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (AFFINE)

Pfizer

Status and phase: Active, not recruiting; Phase 3

Conditions: Hemophilia A

Treatments: Biological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy

Study type: Interventional

Funder types: Industry

Identifiers

NCT04370054

C3731003

2019-004451-37 (EudraCT Number)

Details and patient eligibility

About

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) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have been followed on routine prophylaxis with FVIII products in the Lead-In study C0371004.

Enrollment: 76 patients

Sex: Male

Ages: 18 to 64 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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76 participants in 1 patient group

PF-07055480 (giroctocogene fitelparvovec)

Experimental group

Description:

Single administration of PF-07055480

Treatment:

Biological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy