Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
- Conditions
- Hemophilia A
- Registration Number
- NCT04370054
- Lead Sponsor
- Pfizer
- 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) 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 76
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Total annualized bleeding rate (ABR) 15 months
- Secondary Outcome Measures
Name Time Method Annualized bleeding rate (ABR) 15 months FVIII activity levels 15 months Annualized infusion rate (AIR) of exogenous Factor VIII Activity 15 months Annualized FVIII consumption 15 months Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location 15 months Change in joint health using HJHS (Hemophilia Joint Health Score) 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) 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) 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 5-year study period
Trial Locations
- Locations (36)
NOW Physical Therapy
🇺🇸Mountain View, California, United States
Clinical and Translational Research Unit (CTRU)
🇺🇸Palo Alto, California, United States
Lucile Packard Childrens Hospital
🇺🇸Palo Alto, California, United States
Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
🇺🇸Redwood City, California, United States
UCSF IDS Pharmacy
🇺🇸San Francisco, California, United States
University of California, San Francisco - Clinical Research Center
🇺🇸San Francisco, California, United States
University of California, San Francisco - Moffitt/Long Inpatient Hematology
🇺🇸San Francisco, California, United States
University of California, San Francisco - Outpatient Hematology Clinic
🇺🇸San Francisco, California, United States
Stanford Health Care
🇺🇸Stanford, California, United States
Washington Institute for Coagulation
🇺🇸Seattle, Washington, United States
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