One-Shot gene therapy could free patients from lifelong injections
NCT ID NCT03569891
Summary
This study tested a one-time gene therapy for adults with severe hemophilia B, a genetic bleeding disorder. The therapy uses a modified virus to deliver a corrected gene to the liver, aiming to help the body produce its own missing clotting factor (Factor IX). The goal was to see if this single treatment could reduce or eliminate the need for regular, lifelong infusions of clotting factor medication.
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Contacts and locations
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Locations
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Amsterdam UMC - Locatie AMC
Amsterdam, 1105 AZ, Netherlands
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Bloodworks Northwest
Seattle, Washington, 98104, United States
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Center for Thrombosis and Hemostasis Skåne University Hospital Malmö
Malmo, SE-205 02, Sweden
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Children's National Medical Center Hematology and Oncology
Washington D.C., District of Columbia, 20010, United States
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Cliniques Universitaires Saint-Luc
Brussels, 1200, Belgium
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Erasmus MC
Rotterdam, 3015 CE, Netherlands
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Hemophilia Center of Western New York
Buffalo, New York, 14209, United States
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Klinikum der Johann Wolfgang Goethe Universitat
Frankfurt am Main, 60590, Germany
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Los Angeles Orthopedic Hospital
Los Angeles, California, 90007, United States
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National Coagulation Centre, St James's Hospital
Dublin, D08 A978, Ireland
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Oregon Health & Science University
Portland, Oregon, 97239, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Righospitalet
Copenhagen, 2100, Denmark
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The Cambridge Haemophilia and Thrombophilia Centre Camridge University Hospitals NHS Foundation Trust - Box 217 Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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The Royal London Hospital (Barts Health NHS Trust)
London, E1 2ES, United Kingdom
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UMC Utrecht, Van Creveldkliniek
Utrecht, 3508 GA, Netherlands
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Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
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University Hospital Leuven
Leuven, 3000, Belgium
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University Hospital Southampton NHS Foundation Trust
Southampton, SO16 6YD, United Kingdom
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University of California, Davis
Sacramento, California, 95817, United States
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University of California, San Diego
San Diego, California, 92161, United States
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University of Colorado Denver
Aurora, Colorado, 80045, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of North Carolina, Chapel Hill
Chapel Hill, North Carolina, 27599, United States
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University of South Florida
Tampa, Florida, 33612, United States
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University of Tennessee Health Science Center
Memphis, Tennessee, 38163, United States
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University of Texas Health Science Center & Medical School
Houston, Texas, 77030, United States
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University of Utah
Salt Lake City, Utah, 84108, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
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Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
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Washington Institute for Coagulation
Seattle, Washington, 98101, United States
Conditions
Explore the condition pages connected to this study.