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

Hemophilia A Clinical Trial

— AFFINE  

Official title:

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%)

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
• 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
• 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

Related Conditions & MeSH terms

• Hemophilia A

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

External Links

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