Clinical trial
Cerliponase Alfa Observational Study
Name
190-501
Description
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.
Trial arms
Trial start
2020-08-19
Estimated PCD
2030-08-31
Trial end
2030-08-31
Status
Active (not recruiting)
Treatment
Cerliponase Alfa
Commercially available product provided to patient by participating clinic site.
Arms:
Cerliponase alfa patients
Other names:
Brineura
Administration Kit
Commercially available administration kit provided to the patient by participating clinic site.
Arms:
Cerliponase alfa patients
Size
35
Primary endpoint
Safety surveillance of cerliponase alfa
10 years
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.
Protocol
{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 35, 'type': 'ESTIMATED'}}
Updated at
2024-01-31
1 organization
2 products
1 indication
Organization
BioMarin PharmaceuticalProduct
Cerliponase AlfaProduct
Cerliponase