Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome.
Paul D Ross, Kamal K E Gadalla, Sophie R Thomson...
https://pubmed.ncbi.nlm.nih.gov/40173263Actively 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
33
Participants Needed
16
Research Sites
N/A
Total Duration
On this page
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 brain's ventricles under general anesthesia. Participants receive one administration and will be monitored for 3 years to assess the therapy's 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Single administration
Participants receive a single administration of NGN-401 gene therapy under general anesthesia via intracerebroventricular delivery.
1 treatment visit (in-person)
Follow-up
Duration - 3 years
Participants are followed for efficacy and safety for 3 years after treatment.
Regular follow-up visits over 3 years
Long-term Monitoring
Duration - Up to 12 years
Participants are expected to enroll in a long-term follow-up study for ongoing monitoring for up to 12 years after treatment.
Periodic visits as scheduled in the long-term follow-up study
Trial Site Locations
Total: 16 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
4
Nicklaus Children's Hospital Research Institute
Miami, Florida, United States, 33155
Actively Recruiting
5
Rush University Medical Center
Chicago, Illinois, United States, 60612
Actively Recruiting
6
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Actively Recruiting
7
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
8
Montefiore Medical Center
New York, New York, United States, 10467
Actively Recruiting
9
UNC at Chapel Hill
Chapel Hill, North Carolina, United States, 27514
Actively Recruiting
10
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
11
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
12
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
13
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
14
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
Active, Not Recruiting
15
Royal Hospital for Children and Young People
Edinburgh, United Kingdom, EH16 4TJ
Terminated
16
Manchester University NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
Terminated
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Research Team
C
Contact Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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Published Research Related To This Trial
Preclinical Milestones in MECP2 Gene Transfer for Treating Rett Syndrome.
Indumathy Jagadeeswaran, Jiyoung Oh, Sarah E Sinnett
https://pubmed.ncbi.nlm.nih.gov/38723617