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

Rett Syndrome Clinical Trial

Official title:
A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Pediatric Subjects With Rett Syndrome

Clinical Trial Summary

This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in female children with typical Rett syndrome.

Clinical Trial Description

The study is a phase 1/2, open-label study designed to assess the safety, tolerability, and efficacy of administration of 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 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05898620
Study type Interventional
Source Neurogene Inc.
Contact Contact Center
Phone +1 877-237-5020
Email medicalinfo@neurogene.com
Status Recruiting
Phase Phase 1/Phase 2
Start date June 13, 2023
Completion date October 2029
View Details
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