Experimental gene therapy aims to let platelets produce missing clotting factor
NCT ID NCT03818763
First seen Nov 15, 2025 · Last updated May 14, 2026 · Updated 21 times
Summary
This early-phase study tests a new gene therapy for adults with severe hemophilia A who have or had inhibitors. Doctors take the patient's own stem cells, add a gene that makes Factor VIII (a clotting protein), and return the cells. The goal is to see if this is safe and if the modified cells can produce Factor VIII from platelets. Only 5 participants are enrolled.
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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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Locations
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Froedtert Hospital and the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Conditions
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