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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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Cornell University||Joan & Sanford I. Weill Medical College
RECRUITINGNew York, New York, 10065, United States
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Ege Universitesi Hastanesi
RECRUITINGIzmir, İ̇zmir, 35100, Turkey (Türkiye)
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Kyung Hee University Hospital at Gangdong
NOT_YET_RECRUITINGSeoul, 05278, South Korea
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Mississippi Center For Advanced Medicine
TERMINATEDMadison, Mississippi, 39110, United States
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National Taiwan University Hospital
NOT_YET_RECRUITINGTaipei, 10041, Taiwan
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Royal Prince Alfred Hospital
RECRUITINGCamperdown, New South Wales, 2050, Australia
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Skåne University Hospital
NOT_YET_RECRUITINGMalmö, 21428, Sweden
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Sydney Local Health District (RPAH Zone)
RECRUITINGCamperdown, New South Wales, 2050, Australia
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The Childrens Hospital of Philadelphia Division of Hematology
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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The Regents of the University of California||UC Davis Health
RECRUITINGRancho Cordova, California, 95670, United States
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The University of South Florida Board of Trustees
RECRUITINGTampa, Florida, 33620, United States
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UC Davis Ambulatory Care Clinic
RECRUITINGSacramento, California, 95817, United States
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UC Davis Health
RECRUITINGSacramento, California, 95816, United States
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UC Davis Hemophilia Treatment Center
RECRUITINGSacramento, California, 95817, United States
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UC Davis Medical Center
RECRUITINGSacramento, California, 95817, United States
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UCSF Outpatient Hematology Clinic
NOT_YET_RECRUITINGSan Francisco, California, 94143, United States
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USF Health Morsani Center For Advanced Healthcare
RECRUITINGTampa, Florida, 33612, United States
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Washington Institute for Coagulation
NOT_YET_RECRUITINGSeattle, Washington, 98101, United States
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Weill Cornell Medical College-New York Presbyterian Hospital
RECRUITINGNew York, New York, 10065, United States
Conditions
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