Gene therapy trial targets rare brain disease
NCT ID NCT04680065
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a gene therapy called AAV2-GDNF for multiple system atrophy (MSA), a rare and serious brain disorder. Nine adults with MSA will receive either the gene therapy or a sham surgery. The main goal is to check safety, but researchers will also look for any signs of symptom improvement.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AAV2-GDNF gene therapy
- What this could lead to
- If safe and effective, this could point toward a treatment that slows or improves symptoms of multiple system atrophy.
- What could go wrong
- This is a very early Phase 1 trial with only 9 people, focused on safety. It may not show any benefit, and gene therapy carries risks like inflammation or immune reactions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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
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