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

Overview

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.

Full Title of Study: “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%)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 17, 2024

Interventions

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

Arms, Groups and Cohorts

  • Experimental: PF-07055480 (giroctocogene fitelparvovec)
    • Single administration of PF-07055480

Clinical Trial Outcome Measures

Primary Measures

  • Total annualized bleeding rate (ABR)
    • Time Frame: 15 months

Secondary Measures

  • FVIII activity levels
    • Time Frame: 15 months
  • Annualized bleeding rate (ABR)
    • Time Frame: 15 months
  • Annualized infusion rate (AIR) of exogenous Factor VIII Activity
    • Time Frame: 15 months
  • Annualized FVIII consumption
    • Time Frame: 15 months
  • Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location
    • Time Frame: 15 months
  • Change in joint health using HJHS (Hemophilia Joint Health Score)
    • Time Frame: Yearly up to 5 years
    • HJHS is a validated outcome tool developed for the assessment of joint health in people with hemophilia. The ordinal joint score assesses 9 items in 6 index joints.
  • Patient Reported Outcome (PRO) instrument – Hemophilia Activities List (HAL)
    • Time Frame: Yearly up to 5 years
    • HAL is a disease specific measure of the impact of hemophilia on functional abilities in adults. The instrument consists of 42 items across 7 domains, utilizing a past month recall period. Each item is rated on a scale of 0-6 with higher recoded scores indicating more functional limitations.
  • Patient Reported Outcome (PRO) instrument – Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)
    • Time Frame: Yearly up to 5 years
    • Haem-A-QoL is a disease specific measure of health-related quality of life in patients with hemophilia. Intended for adults, the instrument uses a 4-week recall period to assess health across 10 domains consisting of 46 items. Each item is rated on a scale of 0-6 with lower scores indicating better health-related quality of life.
  • Incidence and severity of AEs
    • Time Frame: 5-year study period

Participating in This Clinical Trial

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

Gender Eligibility: Male

Minimum Age: 18 Years

Maximum Age: 64 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Pfizer
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Pfizer CT.gov Call Center, Study Director, Pfizer

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