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Gene therapy trial targets rare brain disease

NCT ID NCT04680065

Disease control Sponsor: Brain Neurotherapy Bio, Inc. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a gene therapy called AAV2-GDNF for multiple system atrophy (MSA), a rare and serious brain disorder. Nine adults with MSA will receive either the gene therapy or a sham surgery. The main goal is to check safety, but researchers will also look for any signs of symptom improvement.

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 gene therapy
What this could lead to
If safe and effective, this could point toward a treatment that slows or improves symptoms of multiple system atrophy.
What could go wrong
This is a very early Phase 1 trial with only 9 people, focused on safety. It may not show any benefit, and gene therapy carries risks like inflammation or immune reactions.

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

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 9 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2023

Expected to finish

Aug 2028

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

35 to 75 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: * Male and female 35-75 years of age (inclusive) * Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and \> 30 years of age * Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years * Stable anti-parkinsonian medication regimen * Ability to walk a distance of 25 feet with or without an assistive device Exclusion Criteria: * Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases * Presence of dementia, psychosis, substance abuse or poorly controlled depression * Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities * History of cancer or poorly controlled medical conditions that would increase surgical risk * Received investigational agent within 12 weeks * Inability to tolerate laying flat in an MRI and/or allergy to gadolinium NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

multiple system atrophy multiple system atrophy, parkinsonian type

As listed by the trial registrant

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

Multiple system atrophy

Contacts and locations

Locations

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • NYU Langone Health

    New York, New York, 10016, United States

  • Parkinson's Disease and Movement Disorders Center of Boca Raton

    Boca Raton, Florida, 33486, United States

  • Quest Research Institute

    Farmington Hills, Michigan, 48334, United States

  • The Ohio State University Medical Center

    Columbus, Ohio, 43210, United States

  • University of California Irvine

    Irvine, California, 92697, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

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