Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Completed · Phase 1/Phase 2
Conditions studied: Mucopolysaccharidosis IIIB
In brief
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.
Key facts
- Study ID
- NCT02324049
- Run by
- Alexion Pharmaceuticals, Inc.
- People needed
- 11
- Starts
- 2015-01-22
- Expected to finish
- 2017-10-16
- Last updated by the study team
- 2018-08-21
Who can join
Age: 2 and older, up to 12. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Study site — Minneapolis, Minnesota, United States
- Study site — Pittsburgh, Pennsylvania, United States
- Study site — Barcelona, Spain
- Study site — Birmingham, United Kingdom
Full record on ClinicalTrials.gov
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