Status:
WITHDRAWN
An Observational Study in Children With CLN2 Batten Disease
Lead Sponsor:
REGENXBIO Inc.
Conditions:
Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
Eligibility:
All Genders
Brief 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 ...
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 ...
Eligibility Criteria
Inclusion
- A participant is eligible to be included in the study only if all of the following criteria apply:
- His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
- 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
- Is currently receiving biweekly ERT treatment with cerliponase alfa
Exclusion
- A participant is excluded from the study if any of the following criteria apply:
- Has had prior treatment with an adeno-associated virus-based AAV gene therapy
- Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease
Key Trial Info
Start Date :
March 31 2021
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 1 2023
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04462692
Start Date
March 31 2021
End Date
October 1 2023
Last Update
November 1 2021
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