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

Phase 3

Active, not recruiting

• 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

Study Timeline

• Study First Submitted: 2020-04-21
• Study First Submitted QC: 2020-04-29
• Study First Posted: 2020-04-30
• Study Start: 2020-08-18
• Primary Completion: 2024-06-17
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-23
• Last Update Posted: 2024-07-24
• Study Completion: 2028-10-25

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 76

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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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
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
Annualized bleeding rate (ABR)	15 months

Trial Locations

Locations (36)

UCSF IDS Pharmacy; 🇺🇸 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

Hopital Necker; 🇫🇷 Paris, France

Hospital Universitari Vall d'Hebron; 🇪🇸 Barcelona, Spain

Clinical and Translational Research Unit (CTRU); 🇺🇸 Palo Alto, California, United States

NOW Physical Therapy; 🇺🇸 Mountain View, California, United States

Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City; 🇺🇸 Redwood City, California, United States

University of California, San Francisco - Clinical Research Center; 🇺🇸 San Francisco, California, United States

Stanford Health Care; 🇺🇸 Stanford, California, United States

University of Washington Medical Center - Translational Research Unit (TRU); 🇺🇸 Seattle, Washington, United States

Washington Institute for Coagulation; 🇺🇸 Seattle, Washington, United States

Centro de Hematologia e Hemoterapia de Campinas - Hemocentro UNICAMP; 🇧🇷 Campinas, SAO Paulo, Brazil

The Alfred Hospital; 🇦🇺 Melbourne, Victoria, Australia

McMaster University Medical Centre - Hamilton Health Sciences; 🇨🇦 Hamilton, Ontario, Canada

Juravinski Hospital - Hamilton Health Sciences; 🇨🇦 Hamilton, Ontario, Canada

Vivantes Klinikum im Friedrichshain; 🇩🇪 Berlin, Germany

Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II; 🇩🇪 Frankfurt am Main, Germany

General Hospital of Athens "Hippokration"; 🇬🇷 Athens, Greece

General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center; 🇬🇷 Athens, Greece

Università degli studi di Roma "La Sapienza"- Policlinico Umberto I; 🇮🇹 Roma, RM, Italy

Azienda Ospedaliero Universitaria Careggi SODc Malattie Emorragiche e della Coagulazione; 🇮🇹 Firenze, Italy

Dip. di Medicina Clinica e Chirurgia, Università degli Studi di Napoli Federico II - UOC di Medicina; 🇮🇹 Napoli, Italy

Nagoya University Hospital - Transfusion Medicine; 🇯🇵 Nagoya, Aichi, Japan

Kyung Hee University Hospital at Gangdong; 🇰🇷 Seoul, Korea, Republic of

Saitama Medical University Hospital; 🇯🇵 Iruma-gun, Saitama, Japan

H.U. Rio Hortega; 🇪🇸 Valladolid, Spain

Skåne University Hospital; 🇸🇪 Malmö, Skane, Sweden

Adana Acibadem Hospital; 🇹🇷 Adana, Turkey

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan

Ege University Medical Faculty, Pediatric Hematology; 🇹🇷 Izmir, Turkey

Gaziantep University Sahinbey Research and Training Hospital; 🇹🇷 Gaziantep, Turkey

Ege University Medical Faculty; 🇹🇷 Izmir, Turkey

Guy's and St. Thomas' NHS Foundation Trust; 🇬🇧 London, United Kingdom

King Faisal Specialist Hospital & Research Center; 🇸🇦 Riyadh, Saudi Arabia

Lucile Packard Childrens Hospital; 🇺🇸 Palo Alto, California, United States