Gene therapy for hemophilia: how safe is it years later?

NCT ID NCT05568719

Recruiting now Knowledge-focused Sponsor: Pfizer Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hemophilia A hemophilia B Hemorrhage

As listed by the trial registrant

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

HEMOPHILIA A HEMOPHILIA B

Contacts and locations

Locations

Acibadem Adana Hospital
NOT_YET_RECRUITING

Adana, 01130, Turkey (Türkiye)
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
Taichung Veterans General Hospital
NOT_YET_RECRUITING

Taichung, 40705, Taiwan
The Childrens Hospital of Philadelphia Division of Hematology
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
The Childrens Hospital of Philadelphia Division of Hematology
RECRUITING

Philadelphia, Pennsylvania, 19146, 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
Unity Health Toronto, St. Michael's Hospital
NOT_YET_RECRUITING

Toronto, Ontario, M5B 1W8, Canada
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

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