An Observational Study in Children With CLN2 Batten Disease

Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Clinical Trial

Official title:

A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04462692
• Other study ID #: RGX-381-9101
• Status: Withdrawn
• Start date: March 31, 2021
• Est. completion date: October 2023

Study information

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

Clinical Trial Summary

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.

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 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 2023
• Est. primary completion date: October 2023
• 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. 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

Related Conditions & MeSH terms

• Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
• Neuronal Ceroid-Lipofuscinoses

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Regenxbio Inc.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in retinal structure in children with CLN2 Batten disease	As assessed by SD-OCT measures over time.	96 weeks
Secondary	Change in visual function	As assessed by visual acuity over time.	96 weeks
Secondary	Change in visual function	As assessed by pupillary light reflex over time.	96 weeks