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Phase 2 open-label study of intracerebroventricular cerliponase alfa in pediatric CLN2 disease
2015-000891-85Phase 2Unknown
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Mar 2017
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of cerliponase alfa in neuronal ceroid lipofuscinosis type 2 (CLN2), sponsored by BioMarin Pharmaceutical Inc. Primary endpoints: 0 to 6-point motor-language score on the Hamburg CLN2 rating scale and Rate of CLN2 decline. Status: unknown.
02
Drugs
cerliponase alfa
03
Conditions
neuronal ceroid lipofuscinosis type 2 (CLN2)
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