Gene therapy trial hopes to slow Parkinson's in people with GBA1 mutation
NCT ID NCT07011771
First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 23 times
Summary
This early-stage trial tests a single dose of CAP-003 gene therapy in 31 adults with Parkinson's disease who have a specific GBA1 gene mutation. The main goal is to check safety and tolerability over two years, while also measuring changes in a key biomarker linked to the disease. Participants receive the therapy through an IV and attend regular clinic visits for monitoring.
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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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Baylor College of Medicine
Houston, Texas, 77030, United States
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Booth Gardner Parkinson's Care Center - Evergreen Neuroscience Institute
Kirkland, Washington, 98034, United States
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Inland Northwest Research
Spokane, Washington, 99202, United States
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New York Presbyterian Hospital-Columbia University Medical Center
New York, New York, 10032, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Conditions
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