Trial Results

Informed Consent Form
Available Languages: English
Protocol
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Statistical Analysis Plan
Available Languages: English

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

Study Identifier:
NGLU-CL01-T
ClinicalTrials.gov Identifier:
EudraCT Identifier:
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: SBC-103
Date
Oct 2015 - Aug 2017
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 5+ years
Requirements Information
Sex
Female & Male
Age
5+ years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: SBC-103
Date
Oct 2015 - Aug 2017
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 5+ years years
Requirements Information

Protocol 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.

Trial Locations

Location
Status
Location
Birmingham, United Kingdom, B46NH
Status
N/A