Could a single gene fix help stop bleeding in hemophilia a?
NCT ID NCT03818763
First seen Jun 27, 2026 · Last updated Jul 14, 2026 · Updated 2 times
Summary
This early-stage trial tests a gene therapy for adults with severe hemophilia A who have or had Factor VIII inhibitors. Doctors take the patient's own blood stem cells, add a gene that makes Factor VIII, and return the modified cells. The goal is to see if this approach is safe and can help platelets produce the missing clotting protein on their own.
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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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Froedtert Hospital and the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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