NCT02618512

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

A Phase I/II 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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study. The NGLU-CL01 study was a non-interventional study that evaluated structural brain abnormalities and blood brain barrier (BBB) integrity by magnetic resonance imaging (MRI) and cerebrospinal fluid/serum albumin index.

Conditions

• Mucopolysaccharidosis III, Type B (MPS IIIB)
• Sanfilippo B

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2015-10-15
• Primary completion: 2017-08-18
• Completion: 2017-08-18
• First posted: 2015-12-01
• Last updated: 2018-04-17
• Results posted: 2018-04-17

Locations

1 site across 1 country: United Kingdom