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Cerliponase Alfa Observational Study in the US

NCT04476862 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 35

Last updated 2026-01-14

No results posted yet for this study

Summary

This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with cerliponase alfa. Patients receiving or expected to receive cerliponase alfa within 60 days of signing the informed consent form (ICF) may be eligible to enroll in the study, assuming all regulatory requirements for sites that have agreed to participate and protocol inclusion criteria are met. Data may be collected for all or some of the assessments as outlined in the protocol, dependent upon the clinic's and/or individual patient's standard of care.

Conditions

  • Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

Interventions

DRUG

Cerliponase Alfa

Commercially available product provided to patient by participating clinic site.

DEVICE

Administration Kit

Commercially available administration kit provided to the patient by participating clinic site.

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD · BioMarin Pharmaceutical

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-08-19
Primary Completion
2030-08-31
Completion
2030-08-31
FDA Drug
Yes
FDA Device
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

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 NCT04476862 on ClinicalTrials.gov