Brain gene therapy tested for devastating neurological disease
NCT ID NCT04680065
Summary
This is a very early-stage safety study testing a new gene therapy for multiple system atrophy (MSA), a rare and progressive brain disorder. The therapy, called AAV2-GDNF, is delivered directly into a part of the brain called the putamen. The main goal is to see if the treatment is safe and well-tolerated in 9 participants, while also checking for any early signs that it might help control symptoms.
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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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