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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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

Locations

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Booth Gardner Parkinson's Care Center - Evergreen Neuroscience Institute

    Kirkland, Washington, 98034, United States

  • Inland Northwest Research

    Spokane, Washington, 99202, United States

  • New York Presbyterian Hospital-Columbia University Medical Center

    New York, New York, 10032, United States

  • Rush University Medical Center

    Chicago, Illinois, 60612, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

Conditions

Explore the condition pages connected to this study.