A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

PHASE1/PHASE2TerminatedINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

October 15, 2015

Primary Completion Date

August 18, 2017

Study Completion Date

August 18, 2017

Conditions
Mucopolysaccharidosis III, Type B (MPS IIIB)Sanfilippo B
Interventions
DRUG

SBC-103

SBC-103 is a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU). Patients were started with low dose of SBC-103 for 12 weeks and then will escalate to a higher dose.

Trial Locations (1)

B46NH

Birmingham

Sponsors
All Listed Sponsors
lead

Alexion Pharmaceuticals, Inc.

INDUSTRY