Gene therapy for hemophilia: how safe is it years later?
NCT ID NCT05568719
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows 173 people with hemophilia A or B who previously received a single-dose gene therapy in a Pfizer trial. Researchers will track bleeding episodes, clotting factor levels, and any long-term side effects like blood clots or liver issues. The goal is to understand how well the treatment works over time under routine medical care.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
gene therapy (giroctocogene fitelparvovec for hemophilia A, fidanacogene elaparvovec for hemophilia B)
What this could lead to
If successful, this study will confirm whether a single dose of gene therapy can safely control bleeding for years in people with hemophilia.
What could go wrong
This is a follow-up study, not a new treatment test. It only includes people who already received the gene therapy, so results may not apply to everyone. Long-term risks like liver problems or blood clots are being watched.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Acibadem Adana Hospital
NOT_YET_RECRUITINGAdana, 01130, Turkey (Türkiye)
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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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Taichung Veterans General Hospital
NOT_YET_RECRUITINGTaichung, 40705, Taiwan
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The Childrens Hospital of Philadelphia Division of Hematology
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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The Childrens Hospital of Philadelphia Division of Hematology
RECRUITINGPhiladelphia, Pennsylvania, 19146, 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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Unity Health Toronto, St. Michael's Hospital
NOT_YET_RECRUITINGToronto, Ontario, M5B 1W8, Canada
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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