Brain gene therapy aims to boost dopamine in young Parkinson's patients

NCT ID NCT07267065

Not yet recruiting Disease control Sponsor: Krzysztof Bankiewicz Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a gene therapy called AAV2-hAADC in 9 adults with young-onset Parkinson's disease. The therapy delivers a gene directly to two brain regions (putamen and caudate) to help convert levodopa into dopamine more efficiently. The main goal is to check safety, but researchers will also look for signs that 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-hAADC gene therapy
What this could lead to: If successful, this could point toward a new way to control Parkinson's symptoms by helping the brain produce more dopamine from standard medication.
What could go wrong: This is a very early Phase 1 trial with only 9 participants, so safety and effectiveness are not yet known. Brain surgery carries risks like bleeding or infection, and the therapy may not work as hoped.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for EARLY ONSET PARKINSON DISEASE are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Parkinson disease parkinsonian disorder young-onset Parkinson disease

As listed by the trial registrant

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

EARLY ONSET PARKINSON DISEASE PARKINSON'S DISEASE (PD)

Contacts and locations

Show contact details

Locations

The Ohio State University

Columbus, Ohio, 43210, United States

Contact Email: •••••@•••••

More trials for these conditions

Other studies related to the condition(s) this trial covers.