One-Time gene therapy aims to stop bleeding in severe hemophilia
NCT ID NCT04370054
First seen May 27, 2026 · Last updated May 27, 2026
Summary
This study tests a one-time gene therapy (PF-07055480) in 77 adult men with moderately severe to severe hemophilia A. The goal is to see if the treatment can reduce or prevent bleeding episodes compared to their usual factor VIII infusions. Participants receive a single IV dose and are followed for 5 years.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Access Sourcecorp Deliverex (off-site storage)
San Jose, California, 95131, United States
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Adana Acibadem Hospital
Adana, 01130, Turkey (Türkiye)
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Azienda Ospedaliero Universitaria Careggi SODc Malattie Emorragiche e della Coagulazione
Florence, 50134, Italy
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Clinical and Translational Research Unit (CTRU)
Palo Alto, California, 94304, United States
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Dip. di Medicina Clinica e Chirurgia, Università degli Studi di Napoli Federico II - UOC di Medicina
Naples, 80131, Italy
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Ege University Medical Faculty
Izmir, 35100, Turkey (Türkiye)
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Ege University Medical Faculty, Pediatric Hematology
Izmir, 35100, Turkey (Türkiye)
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Gaziantep University Sahinbey Research and Training Hospital
Gaziantep, 27310, Turkey (Türkiye)
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General Hospital of Athens "Hippokration"
Athens, 11527, Greece
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General Hospital of Athens ''Laiko''
Athens, Attikí, 115 27, Greece
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Guy's and St. Thomas' NHS Foundation Trust
London, SE1 7EH, United Kingdom
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H.U. Rio Hortega
Valladolid, 47012, Spain
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Hamilton Health Sciences Corporation
Hamilton, Ontario, L8L 8E7, Canada
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Hopital Necker
Paris, 75015, France
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Hospital Universitari Vall d'Hebron
Barcelona, 08035, Spain
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Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
Redwood City, California, 94063, United States
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Juravinski Hospital - Hamilton Health Sciences
Hamilton, Ontario, L8V 1C3, Canada
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King Faisal Specialist Hospital & Research Center
Riyadh, Saudi Arabia
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Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II
Frankfurt am Main, 60590, Germany
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Kyung Hee University Hospital at Gangdong
Seoul, 05278, South Korea
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Lucile Packard Childrens Hospital
Palo Alto, California, 94304, United States
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McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5, Canada
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NOW Physical Therapy
Mountain View, California, 94040, United States
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Nagoya University Hospital - Transfusion Medicine
Nagoya, Aichi-ken, 466-8560, Japan
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National Taiwan University Hospital
Taipei, 10002, Taiwan
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Regents of The University of California
Oakland, California, 94607, United States
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Saitama Medical University Hospital
Iruma-gun, Saitama, 350-0495, Japan
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Skåne University Hospital
Malmö, Skåne County, 205 02, Sweden
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Stanford Health Care
Stanford, California, 94305, United States
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Stanford Health Care (hospital and IDS pharmacy)
Stanford, California, 94305, United States
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The Alfred Hospital
Melbourne, Victoria, 3004, Australia
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The Board of Trustees of the Leland Stanford Junior University
Redwood City, California, 94063, United States
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UCSF IDS Pharmacy
San Francisco, California, 94143, United States
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UCSF Outpatient Radiology
San Francisco, California, 94143, United States
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UCSF Outpatient Radiology (alternate location)
San Francisco, California, 94143, United States
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UCSF Outpatient Radiology.
San Francisco, California, 94115, United States
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UCSF lnvestigational Drug Service
San Francisco, California, 94143, United States
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Universidade Estadual de Campina
Campina, 13083-970, Brazil
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University of California, San Francisco - Clinical Research Center
San Francisco, California, 94143, United States
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University of California, San Francisco - Moffitt/Long Inpatient Hematology
San Francisco, California, 94143, United States
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University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, 94143, United States
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University of Washington Medical Center - Translational Research Unit (TRU)
Seattle, Washington, 98195, United States
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Università degli studi di Roma "La Sapienza"- Policlinico Umberto I
Roma, RM, 00161, Italy
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Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
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Washington Institute for Coagulation
Seattle, Washington, 98101, United States
Conditions
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