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Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 5

Last updated 2026-04-08

No results posted yet for this study

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.

Conditions

  • Neuronal Ceroid-Lipofuscinoses
  • Neuronal Ceroid Lipofuscinosis CLN2
  • Spinocerebellar Ataxia, Autosomal Recessive 7

Sponsors & Collaborators

  • Children's Hospital of Orange County

    lead OTHER

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-11-01
Primary Completion
2026-12-31
Completion
2026-12-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04098211 on ClinicalTrials.gov