Ignite Creation Date:
2024-05-06 @ 7:05 PM
Last Modification Date:
2024-10-26 @ 3:00 PM
Study NCT ID:
NCT05898620
Status:
RECRUITING
Last Update Posted:
2024-03-13
First Post:
2023-06-01
Brief Title:
A Novel Regulated Gene Therapy NGN-401 Study for Female Children with Rett Syndrome
Sponsor:
Neurogene Inc
Organization:
Neurogene Inc
Study Overview
Official Title:
A Phase 12 Open-Label Clinical Study to Evaluate Safety Tolerability and Efficacy of NGN-401 in Pediatric Subjects with Rett Syndrome
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will evaluate the safety profile of the investigational gene therapy NGN-401 in female children with typical Rett syndrome
Detailed Description:
The study is a phase 12 open-label study designed to assess the safety tolerability and efficacy of administration of an adeno-associated viral vector serotype 9 AAV9 using Neurogenes proprietary transgene regulation technology NGN-401 contains a full-length human MECP2 gene which is designed to express therapeutic levels of the MECP2 protein while avoiding overexpression
The study treatment will be administered under general anesthesia via intracerebroventricular ICV delivery Each participant will be followed for safety and preliminary efficacy for 5 years after treatment and is expected to enroll in a long-term follow-up study for 10 years
The study treatment will be administered under general anesthesia via intracerebroventricular ICV delivery Each participant will be followed for safety and preliminary efficacy for 5 years after treatment and is expected to enroll in a long-term follow-up study for 10 years
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None