Gene therapy aims to slow Huntington's in first human trial
NCT ID NCT04120493
First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 32 times
Summary
This study tests a new gene therapy called AMT-130 for people with early-stage Huntington's disease. The therapy is injected directly into the brain to lower the production of a harmful protein that causes the disease. The study includes 43 adults aged 25 to 65 and is focused on safety and early signs of effectiveness. It is not a cure, but it may help control the disease and slow its progression.
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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.
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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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.