Actively Recruiting
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
Led by Neurogene Inc. · Updated on 2026-03-13
33
Participants Needed
16
Research Sites
337 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
CONDITIONS
Official Title
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Female participants aged 3 years or older
- Diagnosis of typical Rett syndrome with a documented disease-causing mutation in the MECP2 gene
- Stable anti-epileptic drug regimen for at least 12 weeks prior to enrollment
- Participant must be in the post-regression stage of Rett syndrome
- Participant and caregiver must live within a 2-hour drive of the study center for at least 3 months after treatment
- Participant must have never taken 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
- Any medical condition that prevents intracerebroventricular administration, use of anesthetics, or immune suppression required for study procedures
- Other inclusion and exclusion criteria may also apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Research Team
C
Contact Center
CONTACT
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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