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

NCTID	NCT05898620 (View at clinicaltrials.gov)
Description	This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females 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 (Click here for study contact information)
Enrollment Count	33 (ESTIMATED)
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 due to HLH)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	15
Locations	United States,United Kingdom,Australia

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy, 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