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Clinical Trial Report

Gene Therapy Trial Report

Summary

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

NCTID NCT05898620 (View at clinicaltrials.gov)
Description
Indication Rett Syndrome
Compound Name NGN-401 (scAAV9.P546.MECP2)
Sponsor Neurogene Inc.
Funder Type Industry
Status
Recruiting
Enrollment Count 16

Therapy Information

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
Dose 1 1 x 10^15 vg
Dose 2 3 x 10^15 vg
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2023-06-01
Completion Date 2029-10
Last Update 2024-10-28

Participation Criteria

Eligible Age 4 Years - 10 Years
Standard Ages Child
Eligible Sex FEMALE

Locations

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

Regulatory Information

Has US IND True
Recent Updates Selected for START program (6/5/24); Interim data expected 4Q:24

Resources/Links

Patents
  • US9415121B2
Clinical Publications
News and Press Releases
Preclinical Publications