Could a single gene therapy fix severe hemophilia a?
NCT ID NCT03588299
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 27 times
Summary
This early-stage study tests a gene therapy called BAY2599023 in 11 adults with severe hemophilia A. The treatment delivers a healthy copy of the factor VIII gene so the body can produce its own clotting protein. The goal is to find a safe dose that raises clotting factor levels enough to reduce or prevent bleeding episodes.
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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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Contacts and locations
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Locations
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APHP-Hopital Necker Enfants malades
Paris, Île-de-France Region, 75015, France
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Academisch Medisch Centrum (AMC)
Amsterdam, North Holland, 1105 AZ, Netherlands
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Arkansas Children's Hospital - Hematology / Oncology
Little Rock, Arkansas, 72202, United States
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C.S. Mott Children's Hospital - Hematology / Oncology
Ann Arbor, Michigan, 48109, United States
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CHU Rennes - Hopital Pontchaillou
Rennes, Brittany Region, 35033, France
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Erasmus Medisch Centrum
Rotterdam, South Holland, 3015 CE, Netherlands
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Manchester Royal Infirmary
Manchester, Greater Manchester, M13 9WL, United Kingdom
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SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
Sofia, Sofia City Province, 1756, Bulgaria
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UW Health Carbone Cancer Center
Madison, Wisconsin, 53792, United States
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Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
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University Medical Center Utrecht
Utrecht, 3584 CX, Netherlands
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Universitätsklinikum des Saarlandes
Homburg, Saarland, 66421, Germany
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Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
Conditions
Explore the condition pages connected to this study.