Clinical Trials Logo

Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT04480476
Other study ID # RGX-381-9102
Secondary ID
Status Withdrawn
Phase
First received
Last updated
Start date March 31, 2021
Est. completion date April 2022

Study information

Verified date October 2021
Source Regenxbio Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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


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.


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date April 2022
Est. primary completion date April 2022
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: A participant is eligible to be included in the study only if all of the following criteria apply: 1. The participant's legal guardian(s) is(are) willing and able to provide them written, signed informed consent. 2. 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 3. The participant has had one or more eye examinations by an eye care specialist at any time since birth. Exclusion Criteria: No exclusion criteria apply to this study.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Regenxbio Inc.

Outcome

Type Measure Description Time frame Safety issue
Primary Characterize retinal structural changes in children with CLN2 As assessed in by SD-OCT measures in ophthalmic records of children with CLN2 From first available medical chart through informed consent, an average of 10 years
Secondary Characterize changes in visual function. As measured by changes in visual acuity over time in ophthalmic records of children with CLN2. From first available medical chart through informed consent, an average of 10 years