Cerliponase alfa Observational Study

Brief description of study

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. The primary objective of the study is to evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease). The secondary objectives of this study are to further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, serious devicerelated complications, and to evaluate the effects of Grade III or higher serious adverse events (SAEs) on patient performance on the CLN2 clinical rating scale (motor and language domains).

Clinical Study Identifier: s22-01600

Principal Investigator: Nicolas Julian Abreu.

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