clinical trial · NCT04476862
Cerliponase Alfa Observational Study in the US
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2Cerliponase AlfaAdministration Kit
brief 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.
started
Aug 19, 2020
primary completion
Aug 31, 2030
completion
Aug 31, 2030
last updated
Jun 30, 2026
official title
Cerliponase Alfa Observational Study
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol