A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
Research type
Research Study
Full title
A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Pediatric Subjects with Rett Syndrome
IRAS ID
1008196
Contact name
Albena Patroneva
Contact email
Sponsor organisation
Neurogene Inc.
Clinicaltrials.gov Identifier
Research summary
This first-in-human study will test an experimental gene therapy study treatment, NGN-401. NGN-401 is an adeno-associated viral vector serotype 9 (AAV9), using Neurogene's proprietary transgene regulation technology. NGN-401 contains a full-length human MECP2 gene which is designed to express therapeutic levels of the MECP2 protein while avoiding overexpression. The purpose of this research study is to test whether NGN-401 is safe, tolerable and effective in female children who are 4 to 10 years old with a diagnosis of typical Rett syndrome and who have a documented disease-causing mutation in the MECP2 gene. The study treatment will be administered under general anesthesia via intracerebroventricular (ICV) delivery. Each participant will be followed for safety and preliminary efficacy for 5 years after treatment and is expected to enroll in a long-term follow-up study for 10 years.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0705
Date of REC Opinion
15 Nov 2023
REC opinion
Further Information Favourable Opinion