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Phase I/II open-label study of SBC-103 (rhNAGLU) IV in MPS IIIB (Sanfilippo B)
2015-000753-20Phase 2Completed
- Sponsor
- Alexion Pharmaceuticals
- Start
- Aug 2015
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of lesinidase alfa in Sanfilippo syndrome, sponsored by Alexion Pharmaceuticals. Primary endpoint: Assessment of anti-drug antibodies (ADAs). Started Aug 2015; status: completed.
02
Drugs
lesinidase alfa
03
Conditions
Sanfilippo syndrome
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