One-Shot gene therapy could tame hemophilia b bleeding
NCT ID NCT01687608
First seen Mar 26, 2026 · Last updated May 24, 2026 · Updated 7 times
Summary
This study tests a single intravenous dose of gene therapy (AskBio009) in up to 30 adults with hemophilia B, a bleeding disorder. The therapy delivers a working copy of the factor IX gene to help the body produce its own clotting factor. The main goal is to check safety, but researchers will also measure whether bleeding episodes become less frequent and severe.
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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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BloodCenter of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Bloodworks Northwest
Seattle, Washington, 98104, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Boston
Boston, Massachusetts, 022105, United States
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Emory University
Atlanta, Georgia, 30322, United States
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Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Mount Sinai Medical Center
New York, New York, 10029, United States
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Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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The Hemophilia Center, Oregon Health and Science University
Portland, Oregon, 97239, United States
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U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center
Aurora, Colorado, 80045, United States
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University of California Davis Medical Center
Sacramento, California, 95817, United States
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University of California at San Diego Medical Center
San Diego, California, 92103-8651, United States
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University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute
Minneapolis, Minnesota, 55455, United States
Conditions
Explore the condition pages connected to this study.