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Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in MPS IIIB

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Alexion Pharmaceuticals Identifier:
First received: December 15, 2014
Last updated: August 5, 2016
Last verified: August 2016
To evaluate the safety and tolerability of intravenous (IV) administration of SBC 103 in subjects with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.

Condition Intervention Phase
Mucopolysaccharidosis IIIB Drug: SBC-103 Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Resource links provided by NLM:

Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Safety and Tolerability of IV SBC-103 as measured by Physical examination, vital signs, ECG, clinical laboratory tests, concomitant medication and development of anti-drug antibodies. [ Time Frame: Baseline to Week 156 ]

Secondary Outcome Measures:
  • PK/PD Effect of SBC-103 after single and multiple doses, neurocognitive and developmental function, brain structure volumetric assessment, and indices of microstructural integrity. [ Time Frame: Baseline to Week 156 ]

Estimated Enrollment: 9
Study Start Date: December 2014
Estimated Primary Completion Date: July 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SBC-103
There are three parts to the study, for part A and B patients will be enrolled into one of three doses, then in part C randomized into one of the two different higher doses.
Drug: SBC-103
Other Name: recombinant human alpha-N-acetylglucosaminidase (rhNAGLU)


Ages Eligible for Study:   2 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • Patient will be greater than or equal to 2 years of age but less than 12 years of age at the time of informed consent.
  • Definitive diagnosis of MPS IIIB.
  • Documented developmental delay

Key Exclusion Criteria:

  • Received treatment with gene therapy at any time
  • Previous hematopoietic stem cell or bone marrow transplant.
  • Has any internal or non-removable external metal items that may present a safety risk for study assessments that utilize magnetic fields, or any other medical condition or circumstance in which an MRI is contraindicated according to local institutional policy.
  • Known hypersensitivity to eggs.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02324049

United States, Minnesota
Minneapolis, Minnesota, United States
United States, Pennsylvania
Pittsburgh, Pennsylvania, United States
Barcelona, Spain
United Kingdom
Birmingham, United Kingdom
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Responsible Party: Alexion Pharmaceuticals Identifier: NCT02324049     History of Changes
Other Study ID Numbers: NGLU-CL02
Study First Received: December 15, 2014
Last Updated: August 5, 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Alexion Pharmaceuticals:
Mucopolysaccharidosis type IIIB
Sanfilippo Syndrome
Metabolism, Inborn Errors
Metabolic Diseases
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Lysosomal Storage Diseases
Pathologic Processes

Additional relevant MeSH terms:
Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases processed this record on September 19, 2017