Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
NCT ID: NCT04370054
Last Updated: 2025-08-17
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE3
77 participants
INTERVENTIONAL
2020-08-18
2028-10-25
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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PF-07055480 (giroctocogene fitelparvovec)
Single administration of PF-07055480
PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Interventions
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PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Moderately severe to severe hemophilia A (Factor VIII activity \< =1%)
* Suspension of FVIII prophylaxis therapy post study drug infusion
Exclusion Criteria
* 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
18 Years
64 Years
MALE
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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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
University of Washington Medical Center - Translational Research Unit (TRU)
Seattle, Washington, United States
The Alfred Hospital
Melbourne, Victoria, Australia
Centro de Hematologia e Hemoterapia de Campinas - Hemocentro UNICAMP
Campinas, São Paulo, Brazil
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, Canada
Juravinski Hospital - Hamilton Health Sciences
Hamilton, Ontario, Canada
Hopital Necker
Paris, , France
Vivantes Klinikum im Friedrichshain
Berlin, , Germany
Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II
Frankfurt am Main, , Germany
General Hospital of Athens ''Laiko''
Athens, Attikí, Greece
General Hospital of Athens "Hippokration"
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
Florence, , 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-ken, Japan
Saitama Medical University Hospital
Iruma-gun, Saitama, Japan
King Faisal Specialist Hospital & Research Center
Riyadh, , Saudi Arabia
Kyung Hee University Hospital at Gangdong
Seoul, , South Korea
Hospital Universitari Vall d'Hebron
Barcelona, , Spain
H.U. Rio Hortega
Valladolid, , Spain
Skåne University Hospital
Malmo, Skåne County, Sweden
National Taiwan University Hospital
Taipei, , Taiwan
Adana Acibadem Hospital
Adana, , Turkey (Türkiye)
Gaziantep University Sahinbey Research and Training Hospital
Gaziantep, , Turkey (Türkiye)
Ege University Medical Faculty, Pediatric Hematology
Izmir, , Turkey (Türkiye)
Ege University Medical Faculty
Izmir, , Turkey (Türkiye)
Guy's and St. Thomas' NHS Foundation Trust
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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2024-512075-12-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
C3731003
Identifier Type: -
Identifier Source: org_study_id
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