Gene therapy trial hopes to ease Parkinson's symptoms

NCT ID NCT04167540

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-stage trial tests a single dose of a gene therapy called AAV2-GDNF, delivered directly into the brain, for people with Parkinson's disease. The main goal is to check safety in 11 adults aged 35-75. Researchers will also look at whether it improves motor symptoms.

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 (a gene therapy that delivers GDNF protein to brain cells)

What this could lead to

If safe and effective, this could point toward a new way to slow or improve motor symptoms in Parkinson's disease.

What could go wrong

This is a very early Phase 1 trial with only 11 people, focused on safety. It may not show clear benefit, and brain infusion carries risks like bleeding or infection.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

Parkinson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • The Ohio State University Medical Center

    Columbus, Ohio, 43210, United States

  • University of California Irvine

    Irvine, California, 92697, United States

  • University of California San Francisco

    San Francisco, California, 94103, United States