An Observational Study in Children With CLN2 Batten Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2021-03-31

Study Completion Date

2023-10-31

Brief Summary

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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

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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.

Conditions

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Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Keywords

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CLN2, Batten Disease

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

A participant is eligible to be included in the study only if all of the following criteria apply:

1. His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
2. Has documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 with the same mutation as the participant
3. Is currently receiving biweekly ERT treatment with cerliponase alfa

Exclusion Criteria

A participant is excluded from the study if any of the following criteria apply:

1. Has had prior treatment with an adeno-associated virus-based AAV gene therapy
2. Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Other Identifiers

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RGX-381-9101

Identifier Type: -

Identifier Source: org_study_id