A Retrospective, Chart Review Study to Evaluate Ocular Disease Progression in Children With Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

REGENXBIO Inc.0 sitesMarch 31, 2021

ConditionsLate-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
Sponsor: REGENXBIO Inc.
Primary Endpoint: Characterize retinal structural changes in children with CLN2
Status: Withdrawn
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2.

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 retrospective data collection, ocular disease progression in children with a clinical presentation consistent with CLN2. No investigational product is administered in this retrospective, chart review study.

Registry

clinicaltrials.gov

Start Date

March 31, 2021

End Date

April 2022

Last Updated

4 years ago

Study Type

Observational

Sex

All

Investigators

Sponsor

REGENXBIO Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•A participant is eligible to be included in the study only if all of the following criteria apply:
•The participant's legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
•The participant has a documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 disease who has the same CLN2 mutations as the participant
•The participant has had one or more eye examinations by an eye care specialist at any time since birth.