A Retrospective, Natural History Study in Children With CLN2

October 25, 2021 updated by: REGENXBIO Inc.

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

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

Study Overview

Status

Withdrawn

Conditions

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.

Study Type

Observational

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with CLN2 Batten Disease

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Characterize retinal structural changes in children with CLN2
Time Frame: From first available medical chart through informed consent, an average of 10 years
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Characterize changes in visual function.
Time Frame: From first available medical chart through informed consent, an average of 10 years
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

REGENXBIO Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

March 31, 2021

Primary Completion (Anticipated)

April 1, 2022

Study Completion (Anticipated)

April 1, 2022

Study Registration Dates

First Submitted

July 10, 2020

First Submitted That Met QC Criteria

July 17, 2020

First Posted (Actual)

July 21, 2020

Study Record Updates

Last Update Posted (Actual)

November 1, 2021

Last Update Submitted That Met QC Criteria

October 25, 2021

Last Verified

October 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RGX-381-9102

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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