One-Shot gene therapy aims to free patients from bleeding disorder
NCT ID NCT03588299
Summary
This early-stage study is testing a one-time gene therapy called BAY2599023 (DTX201) for adults with severe hemophilia A. The therapy uses a modified virus to deliver a healthy copy of the gene needed to make clotting Factor VIII, with the goal of helping the body produce this protein on its own. Researchers are primarily looking to find a safe and effective dose that can raise Factor VIII levels above a key threshold.
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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
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