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Active Not Recruiting NCT04476862

Cerliponase Alfa Observational Study in the US

Conditions: Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

Sex: All
Healthy volunteers: No
Enrollment: 35
Sponsor: BioMarin Pharmaceutical

Location: Arkansas Children's Hospital Little Rock Arkansas

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.

Eligibility Criteria

Inclusion Criteria: 1. Diagnosed with CLN2 disease. 2. Currently receiving or plan to begin treatment with cerliponase alfa. 3. Written informed consent/assent obtained. Exclusion Criteria: 1\. Currently receiving treatment in another investigational device or drug study.

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT04476862). StuddyBuddy aggregates publicly available trial information.