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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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
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Gene Editing's lasting impact: a 10-Year safety watch
- Can a single gene shot free hemophilia b patients from regular infusions?
- Can a video call replace the clinic for hemophilia pain relief?
- Newborn screening study aims to catch rare diseases at birth
- Monthly shot could free kids with hemophilia from frequent infusions