NCT05898620

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

A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 33 (estimated)
• Sponsor: Neurogene Inc. · Industry
• Sex: Female
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.

Detailed description

The pivotal study, Embolden (TM), is a conversion of the phase 1/2 study and is an open-label, baseline-controlled, multicenter, single-arm study designed to assess the efficacy, safety, and tolerability of administration of NGN401, an adeno-associated viral vector serotype 9 (AAV9) using Neurogene's proprietary transgene regulation technology. NGN-401 contains a full-length human MECP2 gene and is designed to express therapeutic levels of the MeCP2 protein while avoiding overexpression. The study treatment will be given as a single administration under general anesthesia via intracerebroventricular (ICV) delivery. Each participant will be followed for efficacy and safety for 3 years after treatment, and is expected to enroll in a long-term follow-up study for 12 years.

Conditions

• Rett Syndrome

Interventions

Type	Name	Description
GENETIC	NGN-401	NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.

Timeline

• Start date: 2023-06-13
• Primary completion: 2029-12-01
• Completion: 2029-12-01
• First posted: 2023-06-12
• Last updated: 2026-03-13

Locations

16 sites across 3 countries: United States, Australia, United Kingdom

Regulatory

• FDA-regulated drug study