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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, Hayden.Vreugdenhil@seattlechildrens.org

Seattle Children’s complies with applicable federal and other civil rights laws and does not discriminate, exclude people or treat them differently based on race, color, religion (creed), sex, gender identity or expression, sexual orientation, national origin (ancestry), age, disability, or any other status protected by applicable federal, state or local law. Financial assistance for medically necessary services is based on family income and hospital resources and is provided to children under age 21 whose primary residence is in Washington, Alaska, Montana or Idaho.