Brain-Injected gene therapy aims to halt devastating Huntington's disease
NCT ID NCT04120493
Summary
This early-stage trial is testing a one-time gene therapy called AMT-130, which is injected directly into the brain. The goal is to see if lowering the harmful huntingtin protein is safe and can slow the progression of Huntington's disease in 43 people with early symptoms. The study is primarily focused on establishing safety and looking for early signs that the treatment might work.
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Contacts and locations
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Locations
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CenExel Rocky Mountain Clinical Research
Englewood, Colorado, 80113, United States
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Johns Hopkins University
Baltimore, Maryland, 21287, United States
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Ohio State University
Columbus, Ohio, 43210, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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The University of Texas
Houston, Texas, 77030, United States
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University of Alabama at Birmingham
Birmingham, Alabama, 35294-0111, United States
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University of Arizona (Surgical Site Only)
Tucson, Arizona, 85724, United States
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University of California, San Francisco
San Francisco, California, 94158, United States
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University of Michigan Department of Neurology
Ann Arbor, Michigan, 48105, United States
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University of Washington Medical Center
Seattle, Washington, 98195, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
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Virginia Commonwealth University VCU School of Medicine, Department of Neurology
Richmond, Virginia, 23298, United States
Conditions
Explore the condition pages connected to this study.