Cerliponase Alfa Observational Study in the US

Running, not enrolling

Conditions studied: Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

In brief

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.

Key facts

Study ID

NCT04476862

Run by

BioMarin Pharmaceutical

People needed

35

Starts

2020-08-19

Expected to finish

2030-08-31

Last updated by the study team

2026-06-30

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosed with CLN2 disease.
• Currently receiving or plan to begin treatment with cerliponase alfa.
• Written informed consent/assent obtained.

You may not qualify if…

• Currently receiving treatment in another investigational device or drug study.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Children's Hospital of Orange County — Orange, California, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• Children's National Hospital — Washington D.C., District of Columbia, United States
• Advent Health — Orlando, Florida, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Boston Children's Hospital, — Boston, Massachusetts, United States
• Children's Hospital Minnesota — Minneapolis, Minnesota, United States
• NYU Langone Medical Center — New York, New York, United States
• Mt. Sinai School of Medicine — New York, New York, United States
• University of Rochester Medical Center — Rochester, New York, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• University of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States
• Texas Children's Hospital — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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