Gene therapy watch: how do hemophilia treatments hold up over time?

NCT ID NCT05568719

First seen Feb 28, 2026 · Last updated May 13, 2026 · Updated 8 times

Summary

This study follows people with hemophilia A or B who already received a one-time gene therapy (giroctocogene fitelparvovec or fidanacogene elaparvovec) in earlier Pfizer trials. Researchers will monitor long-term safety, including blood clots, liver issues, and inhibitor development, as well as how well the therapy controls bleeding. About 173 participants will be observed during routine care visits.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Cornell University||Joan & Sanford I. Weill Medical College

    RECRUITING

    New York, New York, 10065, United States

  • Ege Universitesi Hastanesi

    RECRUITING

    Izmir, İ̇zmir, 35100, Turkey (Türkiye)

  • Kyung Hee University Hospital at Gangdong

    NOT_YET_RECRUITING

    Seoul, 05278, South Korea

  • Mississippi Center For Advanced Medicine

    TERMINATED

    Madison, Mississippi, 39110, United States

  • National Taiwan University Hospital

    NOT_YET_RECRUITING

    Taipei, 10041, Taiwan

  • Royal Prince Alfred Hospital

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • Skåne University Hospital

    NOT_YET_RECRUITING

    Malmö, 21428, Sweden

  • Sydney Local Health District (RPAH Zone)

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • The Childrens Hospital of Philadelphia Division of Hematology

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • The Regents of the University of California||UC Davis Health

    RECRUITING

    Rancho Cordova, California, 95670, United States

  • The University of South Florida Board of Trustees

    RECRUITING

    Tampa, Florida, 33620, United States

  • UC Davis Ambulatory Care Clinic

    RECRUITING

    Sacramento, California, 95817, United States

  • UC Davis Health

    RECRUITING

    Sacramento, California, 95816, United States

  • UC Davis Hemophilia Treatment Center

    RECRUITING

    Sacramento, California, 95817, United States

  • UC Davis Medical Center

    RECRUITING

    Sacramento, California, 95817, United States

  • UCSF Outpatient Hematology Clinic

    NOT_YET_RECRUITING

    San Francisco, California, 94143, United States

  • USF Health Morsani Center For Advanced Healthcare

    RECRUITING

    Tampa, Florida, 33612, United States

  • Washington Institute for Coagulation

    NOT_YET_RECRUITING

    Seattle, Washington, 98101, United States

  • Weill Cornell Medical College-New York Presbyterian Hospital

    RECRUITING

    New York, New York, 10065, United States

Conditions

Explore the condition pages connected to this study.