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Cerliponase Alfa Observational 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. 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.
Age
All ages
Sex
ALL
Healthy Volunteers
No
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
Start Date
August 19, 2020
Primary Completion Date
August 31, 2030
Completion Date
August 31, 2030
Last Updated
January 14, 2026
35
ESTIMATED participants
Cerliponase Alfa
DRUG
Administration Kit
DEVICE
Lead Sponsor
BioMarin Pharmaceutical
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.
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