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Cerliponase Alfa Observational Study in the US
Sponsor: BioMarin Pharmaceutical
Summary
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.
Official title: Cerliponase Alfa Observational Study
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
35
Start Date
2020-08-19
Completion Date
2030-08-31
Last Updated
2026-01-14
Healthy Volunteers
No
Interventions
Cerliponase Alfa
Commercially available product provided to patient by participating clinic site.
Administration Kit
Commercially available administration kit provided to the patient by participating clinic site.
Locations (16)
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