What is NGN-401 studying for Rett syndrome?

NGN-401 is being studied to evaluate its safety and effectiveness in females with typical Rett syndrome. This gene therapy aims to deliver a functional MECP2 gene to produce therapeutic protein levels. Neurogene Inc. sponsors this trial to better understand its potential benefits and risks.

Who may be eligible for this Rett syndrome clinical trial?

Females aged 3 years and older with a diagnosis of typical Rett syndrome due to a MECP2 gene mutation may be eligible. Participants should be stable on anti-epileptic drugs and in the post-regression stage. Please review eligibility criteria for more details.

Is there any cost to participate in this Rett syndrome clinical trial?

Study-related care, including the gene therapy and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for travel and other costs.

Actively Recruiting

Phase 3

Age: 3Years +

FEMALE

ID05898620

A Baseline-Controlled, Open-Label, Multicenter, Single-Arm Study Evaluating Efficacy, Safety, and Tolerability of NGN-401 Gene Therapy in Females With Rett Syndrome

Led by Neurogene Inc. · Updated on 2026-03-13

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of an investigational gene therapy called NGN-401 in females diagnosed with typical Rett syndrome. This pivotal phase 3 study, named Embolden, is an open-label and single-arm trial conducted at multiple centers. NGN-401 uses a viral vector to deliver a full-length human MECP2 gene designed to produce therapeutic levels of MeCP2 protein while avoiding overexpression. The study treatment involves a single dose of NGN-401 delivered directly into the brains ventricles under general anesthesia. Participants receive one administration and will be monitored for 3 years to assess the therapys effects. After this period, they are expected to join a long-term follow-up study lasting 12 years. Different dosing levels are planned for various age groups starting from 3 years old. Participants will have regular assessments to monitor efficacy and safety throughout the study. Caregivers and participants must live within a two-hour drive of the study center for at least three months post-treatment. The main outcome measured is the efficacy of NGN-401 after 52 weeks. Researchers will also observe and document any side effects or tolerability issues during the study and long-term follow-up.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 3Years +

FEMALE

Eligibility Criteria

You may qualify if you...

Female participants aged 3 years and older
Diagnosis of typical Rett syndrome with a documented disease-causing MECP2 gene mutation
Stable anti-epileptic drug regimen for at least 12 weeks
Participant is in the post-regression stage of Rett syndrome
Participant and caregiver reside within a 2-hour drive of the study center for at least 3 months after treatment
No prior use of trofinetide, or discontinued it due to tolerability, lack of efficacy, or other reasons

You will not qualify if you...

Normal or near normal hand function
Presence of any clinically significant condition other than Rett syndrome
Medical conditions that prevent intracerebroventricular administration, use of anesthetics, or required immune suppression
Other unspecified inclusion and exclusion criteria may apply

Research Team

Contact Center

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