Gene therapy trial offers hope for hemophilia b patients
NCT ID NCT00979238
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This 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, aiming to reduce bleeding episodes. Fourteen participants received one of four doses to check safety and how well the therapy works.
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the original study
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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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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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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