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NGN-401 Gene Therapy for Rett Syndrome

This study is testing a new gene therapy called NGN-401 for females with Rett syndrome. NGN-401 is designed to deliver a working copy of the MECP2 gene, which is faulty in Rett syndrome, to help the body produce the necessary protein. The study aims to see how well NGN-401 works and if it is safe. To join, you must be a female with a confirmed diagnosis of typical Rett syndrome due to a MECP2 gene mutation, and be at least 3 years old. The study is currently recruiting participants for Arm 4. Success will be measured by how well NGN-401 works after 52 weeks.

Study design
This is an open-label (meaning everyone knows what treatment is given), single-arm study, which means all participants receive the same treatment. It plans to enroll 33 participants.
What's involved
The study treatment, NGN-401, is given once as a single injection into the fluid surrounding the brain and spinal cord (intracerebroventricular or ICV delivery) while under general anesthesia.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 3 years after treatment to check for effectiveness and safety, and are expected to join a long-term follow-up study for 12 years.

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NCT05898620

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

Active, Not Recruiting
PHASE3Ages 3+InterventionalTreatment
Neurogene Inc.
~33 participants
Updated 2026-06-10 on ClinicalTrials.gov
What's tested:NGN-401

At a glance

Recruiting sites
0 of 16 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Efficacy of NGN-401
Measured over 52 Weeks

Conditions

Rett Syndrome

Explore related trials

Rett SyndromeMECP2 Gene

Where it's being run

16 sites across 15 states
United Kingdom2
Alabama1
California1
Colorado1
Florida1
Illinois1
Maryland1
Massachusetts1

Study leadership

  • Julie Jordan, MD · STUDY_DIRECTOR · Neurogene Inc.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Efficacy of NGN-40152 Weeks

    Responders will be defined as participants who: * Attain a CGI-I score of ≤ 3 ("minimally improved"); * and gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters.