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Gene therapy trial aims to help girls with rett syndrome gain new skills

NCT ID NCT05898620

Disease control Sponsor: Neurogene 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whether participants show any improvement and gain at least one new milestone from a list of 28 skills.

What this could mean

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

Active substance
NGN-401 (a gene therapy using a harmless virus to deliver a working copy of the MECP2 gene)
What this could lead to
If successful, this could become a treatment that helps girls with Rett syndrome gain new skills and improve their daily lives.
What could go wrong
This is an early-stage, small trial (33 people) with no placebo group, so results may not be definitive. Gene therapies can have side effects like immune reactions or liver issues.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 33 people

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

Started

Jun 2023

Expected to finish

Dec 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

3 years and older

Sex

Female participants only

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: * Females who are between the ages of ≥4 and ≤10 years for Arms 1 and 2 (Arms closed). Females who are ≥11 years of age or older for Arm 3 (Arm closed). Females who are ≥3 for Arm 4, the pivotal cohort. * Diagnosis of typical Rett syndrome with a documented disease-causing mutation in the methyl-CpG-binding protein 2 (MECP2) gene * Current anti-epileptic drug regimen has been stable for at least 12 weeks * Participant must be in the post-regression stage * Participant and caregiver should reside within a 2-hour drive of the study center for at least 3 months following treatment * Participant must have never taken trofinetide or have taken trofinetide and discontinued due to tolerability, lack of efficacy, or other reasons. Following NGN-401 dosing, trofinetide may be initiated after a specified time period and with the support of the treating clinician. Exclusion Criteria: * Normal or near normal hand function * Has a current clinically significant condition other than Rett syndrome * Presence of a concomitant medical condition that precludes intracerebroventricular administration, or use of anesthetics or immune suppression needed for study related procedures Other inclusion and exclusion criteria apply.

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Conditions

The condition(s) this trial relates to.

hereditary disease intellectual disability nervous system disorder Neurobehavioral Manifestations neurodevelopmental disorder Neurologic Manifestations Pathologic Processes Rett syndrome X-linked disease

As listed by the trial registrant

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

Rett syndrome

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL, United Kingdom

  • Montefiore Medical Center

    New York, New York, 10467, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nicklaus Children's Hospital Research Institute

    Miami, Florida, 33155, United States

  • Royal Hospital for Children and Young People

    Edinburgh, EH16 4TJ, United Kingdom

  • Rush University Medical Center

    Chicago, Illinois, 60612, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • The Children's Hospital at Westmead

    Sydney, New South Wales, Australia

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • UNC at Chapel Hill

    Chapel Hill, North Carolina, 27514, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

More trials for these conditions

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