drugset
drugset / Trial / NCT02324049

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049 ↗

Phase 1/2 Completed 11 enrolled Alexion Pharmaceuticals, Inc.
NaSingle-groupOpen-labelTreatment

Summary

Study to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in participants with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.

Timeline

Start
2015-01-22
Primary completion
2017-10-16
Completion
2017-10-16

Drugs

EvaluationDrugModalityDoseRoute
Subject LESINIDASE ALFA Protein / enzyme biologic 0.3 mg/kg Intravenous
Subject LESINIDASE ALFA Protein / enzyme biologic 1 mg/kg Intravenous
Subject LESINIDASE ALFA Protein / enzyme biologic 3 mg/kg Intravenous
Subject LESINIDASE ALFA Protein / enzyme biologic 5 mg/kg Intravenous
Subject LESINIDASE ALFA Protein / enzyme biologic 10 mg/kg Intravenous

Indications

mucopolysaccharidosis type 3B