Clinical Trial

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

Active, Not Recruiting
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Summary
The purpose of this study is to gather information on the possible symptoms that patients with atypical neuronal ceroid lipofuscinosis type 2 (also known as aTPP1 or atypical tripeptidyl peptidase deficiency) have and how they change over time.
Protocol Amendment History 7 amendments
This ClinicalTrials.gov record has been amended 7 times since 2019-09-18; most recent amendment 2026-04-02.
Status change: Recruiting → Active, Not Recruiting 2021-07-14
Trial Details
NCT Number NCT04098211
Lead Sponsor Children's Hospital of Orange County
Conditions Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Enrollment 5 participants
Start Date 2019-11-01
Primary Completion 2026-12 (estimated)
Study Completion 2026-12 (estimated)
Updated on ClinicalTrials.gov 2026-04-08