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Can a single gene therapy calm Parkinson's for years?

NCT ID NCT05894343

Disease control Sponsor: MeiraGTx, LLC Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 01, 2026 · Last updated Sep 02, 2026 · Updated 1 time

Summary

This study follows people with Parkinson's disease who previously received AAV-GAD, a gene therapy infused into a brain region that helps control movement. The goal is to check how safe the treatment remains over many years, watching for any drug-related side effects. Researchers track participants who took part in an earlier trial to see if the therapy's effects last and whether any long-term problems emerge.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
AAV-GAD, a gene therapy delivered directly into the brain's subthalamic nuclei
What this could lead to
If this gene therapy proves safe over the long term, it could become a lasting treatment option for Parkinson's disease, potentially reducing symptoms with a single procedure.
What could go wrong
This is an early-stage study with only 13 participants, so results may not apply to everyone. Gene therapy carries risks like brain inflammation or side effects from the procedure itself.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

13 people

The number who actually took part.

Started

May 2023

Expected to finish

Oct 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

25 to 86 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Previously enrolled in Study MGT-GAD-025. Exclusion Criteria: * None

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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.

Parkinson's disease

Contacts and locations

Locations

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Henry Ford West Bloomfield Hospital

    West Bloomfield, Michigan, 48322, United States

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107, United States

  • Weill Cornell Medicine

    New York, New York, 10021, United States

  • West Virginia University

    Morgantown, West Virginia, 26506, United States

  • Yale University

    New Haven, Connecticut, 06510, United States

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