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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04370054
Other study ID # C3731003
Secondary ID 2019-004451-37
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date August 18, 2020
Est. completion date October 25, 2028

Study information

Verified date March 2024
Source Pfizer
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 76
Est. completion date October 25, 2028
Est. primary completion date June 17, 2024
Accepts healthy volunteers No
Gender Male
Age group 18 Years to 64 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
Australia The Alfred Hospital Melbourne Victoria
Brazil Centro de Hematologia e Hemoterapia de Campinas - Hemocentro UNICAMP Campinas SAO Paulo
Canada Juravinski Hospital - Hamilton Health Sciences Hamilton Ontario
Canada McMaster University Medical Centre - Hamilton Health Sciences Hamilton Ontario
France Hopital Necker Paris
Germany Vivantes Klinikum im Friedrichshain Berlin
Germany Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II Frankfurt am Main
Greece General Hospital of Athens "Hippokration" Athens
Greece General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center Athens
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 Università degli studi di Roma "La Sapienza"- Policlinico Umberto I Roma RM
Japan Saitama Medical University Hospital Iruma-gun Saitama
Japan Nagoya University Hospital - Transfusion Medicine Nagoya Aichi
Korea, Republic of Kyung Hee University Hospital at Gangdong Seoul
Saudi Arabia King Faisal Specialist Hospital & Research Center Riyadh
Spain Hospital Universitari Vall d'Hebron Barcelona
Spain H.U. Rio Hortega Valladolid
Sweden Skåne University Hospital Malmö Skane
Taiwan National Taiwan University Hospital Taipei
Turkey Adana Acibadem Hospital Adana
Turkey Gaziantep University Sahinbey Research and Training Hospital Gaziantep
Turkey Ege University Medical Faculty Izmir
Turkey Ege University Medical Faculty, Pediatric Hematology Izmir
United Kingdom Guy's and St. Thomas' NHS Foundation Trust London
United States 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 University of Washington Medical Center - Translational Research Unit (TRU) Seattle Washington
United States Washington Institute for Coagulation Seattle Washington
United States Stanford Health Care Stanford California

Sponsors (1)

Lead Sponsor Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  Canada,  France,  Germany,  Greece,  Italy,  Japan,  Korea, Republic of,  Saudi Arabia,  Spain,  Sweden,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Total annualized bleeding rate (ABR) 15 months
Secondary FVIII activity levels 15 months
Secondary Annualized bleeding rate (ABR) 15 months
Secondary Annualized infusion rate (AIR) of exogenous Factor VIII Activity 15 months
Secondary Annualized FVIII consumption 15 months
Secondary Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location 15 months
Secondary Change in joint health using HJHS (Hemophilia Joint Health Score) 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. Yearly up to 5 years
Secondary Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL) 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. Yearly up to 5 years
Secondary Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) 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. Yearly up to 5 years
Secondary Incidence and severity of AEs 5-year study period
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