Gene therapy trial offers hope for hemophilia b patients
NCT ID NCT00979238
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 27 times
Summary
This early-stage study tested a new gene therapy for adults with severe hemophilia B, a bleeding disorder caused by a faulty gene. The treatment uses a harmless virus to deliver a working copy of the factor IX gene into the body. The main goal was to check safety in 14 participants, not yet to prove it works as a cure.
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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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Locations
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Katharine Dormandy Haemophilia Centre and Haemostasis Unit, University College of London
London, United Kingdom
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Scott and White Memorial Hospital
Temple, Texas, 76508, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38119, United States
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Stanford Medical School
Stanford, California, 94305, United States
Conditions
Explore the condition pages connected to this study.