Current Research Studies

Cerliponase alfa Observation Study

Cerliponase Alfa Observational Study

Phase: IV
Clinicaltrials.gov ID: NCT04476862

What is the goal of the 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 long-term safety of cerliponase alfa in patient with CLN2 disease.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team:

PI (Study Doctor): Emily Shelkowitz
Study Team Member: Hayden Vreugdenhil, [email protected]