https://clinicaltrials.gov/ct2/show/NCT04476862
Brineura (cerliponase alfa)
Brief 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.
Condition or disease Intervention/treatment
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Drug: Cerliponase Alfa
Device: Administration Kit
Study Design
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Study Type : Observational
Estimated Enrollment : 35 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Cerliponase Alfa Observational Study
Estimated Study Start Date : August 24, 2020
Estimated Primary Completion Date : August 24, 2030
Estimated Study Completion Date : August 24, 2030
