One-Time brain injection aims to slow Huntington's disease
NCT ID NCT04120493
First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 31 times
Summary
This study tests a new gene therapy called AMT-130 for people with early-stage Huntington's disease. It involves a one-time injection into the brain to lower the production of the harmful huntingtin protein. The trial includes 43 participants and is focused on safety and whether the treatment shows any signs of slowing the disease. While promising, this is an early-stage study, and participants will need ongoing monitoring.
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This is a summary of
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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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.