Ignite Creation Date:
2025-12-24 @ 3:14 PM
Ignite Modification Date:
2025-12-25 @ 1:37 PM
Study NCT ID:
NCT04462692
Status:
WITHDRAWN
Last Update Posted:
2021-11-01
First Post:
2020-07-02
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
An Observational Study in Children With CLN2 Batten Disease
Sponsor:
REGENXBIO Inc.
Organization:
Study Overview
Official Title:
A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease
Status:
WITHDRAWN
Status Verified Date:
2021-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
study stopped due to company decision
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.
Detailed Description:
CLN2 is a rare disease with limited available ocular natural history data. While current standard of care slows motor degeneration, it is not known to treat the ocular manifestations of disease. This study is planned to document, through prospective data collection, ocular disease progression in children with a clinical presentation consistent with CLN2 Batten disease undergoing current standard of care for their condition. No investigational product is administered in this observational study.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
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?: