Ignite Creation Date:
2024-05-06 @ 5:10 PM
Last Modification Date:
2024-10-26 @ 2:24 PM
Study NCT ID:
NCT05228145
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-09-18
First Post:
2021-12-17
Brief Title:
Gene Therapy Study for Children With CLN5 Batten Disease
Sponsor:
Neurogene Inc
Organization:
Neurogene Inc
Study Overview
Official Title:
A Phase 12 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis NCL Subtype 5 CLN5 Disease
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-08
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:
CLN5-200
Brief Summary:
This is a prospective non-randomized open-label dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Neuronal Ceroid Lipofuscinosis Batten Subtype 5 CLN5 disease
Detailed Description:
The study is a first in human FIH open-label dose escalation study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 AAV9 carrying the gene encoding human ceroid-lipofuscinosis neuronal protein 5 CLN5 in subjects with CLN5 Batten disease The study treatment will be delivered via intracerebroventricular ICV and intravitreal IVT injection on the same day Each participant will be followed for safety and efficacy for 5 years after treatment Efficacy assessments in this study will evaluate motor language visual and cognitive function
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