Gene therapy takes on rare brain disease in first human safety trial
NCT ID NCT04680065
First seen Jan 31, 2026 · Last updated May 14, 2026 · Updated 13 times
Summary
This early-stage study tests whether a gene therapy called AAV2-GDNF is safe for people with Multiple System Atrophy (MSA), a rare and serious brain disorder. Nine adults aged 35-75 with MSA will receive either the gene therapy or a placebo through a brain injection. The main goal is to check for side effects, while also looking at whether the treatment might slow the disease.
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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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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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NYU Langone Health
New York, New York, 10016, United States
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Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486, United States
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Quest Research Institute
Farmington Hills, Michigan, 48334, United States
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The Ohio State University Medical Center
Columbus, Ohio, 43210, United States
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University of California Irvine
Irvine, California, 92697, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Conditions
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