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Brain gene therapy tested for Parkinson's – early results in

NCT ID NCT05603312

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-stage trial tested a gene therapy for Parkinson's disease. The treatment involved delivering a gene called GAD into a brain region that controls movement. Only 14 people took part, and the main goal was to check safety, not whether it works. The study is now complete.

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 (gene therapy)
What this could lead to
If successful, this could point toward a new way to control Parkinson's symptoms by delivering a gene that helps calm overactive brain signals.
What could go wrong
This was a very small, early-phase trial focused on safety, not effectiveness. The gene therapy requires brain surgery, which carries risks like infection or bleeding. It may not improve symptoms.

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

14 people

The number who actually took part.

Started

Oct 2022

Finished

Sep 2024

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 85 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: * Confirmed Parkinson's Disease * Levodopa responsiveness for at least 12 months * UPDRS Part 3 score of ≥25 points in the "off" state Exclusion Criteria: * History of brain surgery to treat Parkinson's Disease * Any history of cerebral insult or central nervous system infection * Atypical Parkinson's Disease * Focal or lateralized neurologic deficits * Evidence of significant medical or psychiatric disorders * Cognitive impairment as defined by the Montreal Cognitive Assessment (MoCA) ≤ 20 * Beck Depression Inventory-II score of ≥ 20

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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 - Yale New Haven Hospital

    New Haven, Connecticut, 06510, United States

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