Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Clinical Trial
Official title:
A Retrospective, Chart Review Study to Evaluate Ocular Disease Progression in Children With Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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Status | Withdrawn |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 |
Verified date | October 2021 |
Source | Regenxbio Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2.
Status | Withdrawn |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. |
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Regenxbio Inc. |
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 |