NCT04476862

Cerliponase Alfa Observational Study in the US

Cerliponase Alfa Observational Study

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 35 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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

Type	Name	Description
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.

Timeline

• Start date: 2020-08-19
• Primary completion: 2030-08-31
• Completion: 2030-08-31
• First posted: 2020-07-20
• Last updated: 2026-01-14

Locations

16 sites across 1 country: United States

Regulatory

• FDA-regulated drug study
• FDA-regulated device study