Brain gene therapy aims to silence Huntington's disease
NCT ID NCT04120493
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a one-time gene therapy called AMT-130, injected directly into the brain, to lower the production of the harmful huntingtin protein. It involves 43 adults with early Huntington's disease. The study is primarily checking safety, but also looks for signs that the treatment might slow the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AMT-130 (a gene therapy injected into the brain to lower the harmful huntingtin protein)
What this could lead to
If successful, this could point toward a treatment that slows or stops Huntington's disease progression by reducing the toxic protein at its source.
What could go wrong
This is an early, first-in-human study with only 43 participants, so safety and effectiveness are not yet proven. Brain surgery carries risks like infection, bleeding, or swelling.
Disclaimer
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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