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One-Time gene therapy aims to stop bleeding in severe hemophilia

NCT ID NCT04370054

Ongoing Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 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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Conditions

The condition(s) this trial relates to.

hemophilia A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHILIA A

Contacts and locations

Locations

  • Access Sourcecorp Deliverex (off-site storage)

    San Jose, California, 95131, United States

  • Adana Acibadem Hospital

    Adana, 01130, Turkey (Türkiye)

  • Azienda Ospedaliero Universitaria Careggi SODc Malattie Emorragiche e della Coagulazione

    Florence, 50134, Italy

  • Clinical and Translational Research Unit (CTRU)

    Palo Alto, California, 94304, United States

  • Dip. di Medicina Clinica e Chirurgia, Università degli Studi di Napoli Federico II - UOC di Medicina

    Naples, 80131, Italy

  • Ege University Medical Faculty

    Izmir, 35100, Turkey (Türkiye)

  • Ege University Medical Faculty, Pediatric Hematology

    Izmir, 35100, Turkey (Türkiye)

  • Gaziantep University Sahinbey Research and Training Hospital

    Gaziantep, 27310, Turkey (Türkiye)

  • General Hospital of Athens "Hippokration"

    Athens, 11527, Greece

  • General Hospital of Athens ''Laiko''

    Athens, Attikí, 115 27, Greece

  • Guy's and St. Thomas' NHS Foundation Trust

    London, SE1 7EH, United Kingdom

  • H.U. Rio Hortega

    Valladolid, 47012, Spain

  • Hamilton Health Sciences Corporation

    Hamilton, Ontario, L8L 8E7, Canada

  • Hopital Necker

    Paris, 75015, France

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035, Spain

  • Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City

    Redwood City, California, 94063, United States

  • Juravinski Hospital - Hamilton Health Sciences

    Hamilton, Ontario, L8V 1C3, Canada

  • King Faisal Specialist Hospital & Research Center

    Riyadh, Saudi Arabia

  • Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II

    Frankfurt am Main, 60590, Germany

  • Kyung Hee University Hospital at Gangdong

    Seoul, 05278, South Korea

  • Lucile Packard Childrens Hospital

    Palo Alto, California, 94304, United States

  • McMaster University Medical Centre - Hamilton Health Sciences

    Hamilton, Ontario, L8N 3Z5, Canada

  • NOW Physical Therapy

    Mountain View, California, 94040, United States

  • Nagoya University Hospital - Transfusion Medicine

    Nagoya, Aichi-ken, 466-8560, Japan

  • National Taiwan University Hospital

    Taipei, 10002, Taiwan

  • Regents of The University of California

    Oakland, California, 94607, United States

  • Saitama Medical University Hospital

    Iruma-gun, Saitama, 350-0495, Japan

  • Skåne University Hospital

    Malmö, Skåne County, 205 02, Sweden

  • Stanford Health Care

    Stanford, California, 94305, United States

  • Stanford Health Care (hospital and IDS pharmacy)

    Stanford, California, 94305, United States

  • The Alfred Hospital

    Melbourne, Victoria, 3004, Australia

  • The Board of Trustees of the Leland Stanford Junior University

    Redwood City, California, 94063, United States

  • UCSF IDS Pharmacy

    San Francisco, California, 94143, United States

  • UCSF Outpatient Radiology

    San Francisco, California, 94143, United States

  • UCSF Outpatient Radiology (alternate location)

    San Francisco, California, 94143, United States

  • UCSF Outpatient Radiology.

    San Francisco, California, 94115, United States

  • UCSF lnvestigational Drug Service

    San Francisco, California, 94143, United States

  • Universidade Estadual de Campina

    Campina, 13083-970, Brazil

  • University of California, San Francisco - Clinical Research Center

    San Francisco, California, 94143, United States

  • University of California, San Francisco - Moffitt/Long Inpatient Hematology

    San Francisco, California, 94143, United States

  • University of California, San Francisco - Outpatient Hematology Clinic

    San Francisco, California, 94143, United States

  • University of Washington Medical Center - Translational Research Unit (TRU)

    Seattle, Washington, 98195, United States

  • Università degli studi di Roma "La Sapienza"- Policlinico Umberto I

    Roma, RM, 00161, Italy

  • Vivantes Klinikum im Friedrichshain

    Berlin, 10249, Germany

  • Washington Institute for Coagulation

    Seattle, Washington, 98101, United States

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