drugset

drugset / Trial / NCT05898620

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

NCT05898620 ↗

Phase 3 Active not recruiting 33 enrolled Neurogene Inc.

Non-randomizedSequentialSingle-blindTreatment

Summary

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

Timeline

Start: 2023-06-13
Primary completion: 2029-12
Completion: 2029-12

Publications

Ross PD, Gadalla KKE, Thomson SR, Selfridge J, Bahey NG, Benito J, Burstein SR, McMinn R, Bolon B, Hector RD, Cobb SR. Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome. Sci Transl Med. 2025 Apr 2;17(792):eadq3614. doi: 10.1126/scitranslmed.adq3614. Epub 2025 Apr 2.
Jagadeeswaran I, Oh J, Sinnett SE. Preclinical Milestones in MECP2 Gene Transfer for Treating Rett Syndrome. Dev Neurosci. 2025;47(2):147-156. doi: 10.1159/000539267. Epub 2024 May 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	NGN-401	Unknown	1e+15 vg	Intrathecal
Subject	NGN-401	Unknown	3e+15 vg	Intrathecal

Indications