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

NCTID	NCT05898620 (View at clinicaltrials.gov)
Description	This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in female children with typical Rett syndrome. (Show More)
Development Status	Active
Indication	Rett Syndrome
Disease Ontology Term	DOID:1206
Compound Name	NGN-401
Compound Description	scAAV9.P546.MECP2
Sponsor	Neurogene Inc.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	16
Results Posted	Not Available

Target Gene/Variant	MECP2
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracerebroventricular
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	1E15 vg
Dose 2	3E15 vg (discontinued after patient death)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	8
Locations	United States,United Kingdom,Australia

Has US IND	True
FDA Designations	Support for Clinical Trials Advancing Rare Disease Therapeutics
Recent Updates	Interim data expected 2H:2025; SAE reported for 3E15 vg dose, development will continue using 1E15 vg dose only