Cerliponase alfa Observational Study
Research type
Research Study
Full title
Cerliponase alfa Observational Study
IRAS ID
268183
Contact name
Paul Gissen
Contact email
Sponsor organisation
BioMarin Pharmaceuticals, Inc
Duration of Study in the UK
10 years, 2 months, 1 days
Research summary
CLN2 disease, a form of Batten disease, is an inherited disorder that affects the nervous system. The signs and symptoms of this condition typically begin between ages 2 and 4. Patients with CLN2 disease typically present initially with seizures and a history of language delay, followed by ataxia, myoclonus, impaired speech, cognitive impairment, and developmental regression.
Cerliponase alfa is administered by infusion via a surgically implanted intraventricular reservoir and catheter. Cerliponase alfa was approved by the European Medicines Agency in 2017.
The purpose of this voluntary observational study is to evaluate the long-term safety of cerliponase alfa, and to further assess the occurrence of serious hypersensitivity reactions, serious cardiovascular adverse events, and serious device-related complications; and, to evaluate the
effects of Grade III serious adverse events (SAEs) on patient performance on the CLN2 Clinical Rating Scale (Motor and Language domains).Investigators are encouraged to follow the Recommended Schedule of Events, which includes assessments to monitor the long-term safety of CLN2 patients who are currently being treated, or who plan to be treated with cerliponase alfa within 60 days of signing the ICF/PIAF. Data may be collected for all or some of the assessments dependent upon the individual’s standard of care.
The study duration is up to 10 years. Patients will be enrolled in the study over a period of at least 8 years from the date the first patient is consented.
It is estimated that approximately 45-63 patients will be enrolled in the study.This study is sponsored by BioMarin.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1611
Date of REC Opinion
15 Nov 2019
REC opinion
Further Information Favourable Opinion