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Brineura in Pediatric CLN2 Disease
NCT02678689Phase 2Completed
- Sponsor
- BioMarin Pharmaceutical
- Start
- Jan 2016
- Primary completion
- Apr 2022
- Modality
- Replacement Enzymes
- Countries
- 4
01
Overview
Phase 2, open-label, single-arm trial of cerliponase alfa in neuronal ceroid lipofuscinosis type 2 (CLN2), sponsored by BioMarin Pharmaceutical. Primary endpoints: Rate of Decline in Motor Language Scale Score at 169 weeks and Probability of Unreversed 2-Point Decline in Motor-language Score at 169 weeks. Started Jan 2016; status: completed.
02
Drugs
cerliponase alfa
03
Conditions
neuronal ceroid lipofuscinosis type 2 (CLN2)
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