NCT04476862
CLN2, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
Any
No
Cerliponase Alfa, Administration Kit
Observational
35
2020-08-19
2024-09-19
Little Rock, Arkansas, United States
Orange, California, United States
Aurora, Colorado, United States
Washington, District of Columbia, United States
OrLando, Florida, United States
Atlanta, Georgia, United States
Chicago, Illinois, United States
Boston, Massachusetts, United States
Minneapolis, Minnesota, United States
New York, New York, United States
New York, New York, United States
Rochester, New York, United States
Columbus, Ohio, United States
Oklahoma City, Oklahoma, United States
Houston, Texas, United States
Seattle, Washington, United States
1. Diagnosed with CLN2 disease.
2. Currently receiving or plan to begin treatment with cerliponase alfa.
3. Written informed consent/assent obtained.
1. Currently receiving treatment in another investigational device or drug study
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