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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02324049
Recruitment Status : Active, not recruiting
First Posted : December 24, 2014
Last Update Posted : October 11, 2017
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
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 or disease Intervention/treatment Phase
Mucopolysaccharidosis IIIB Drug: SBC-103 Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
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
Study Start Date : December 2014
Estimated Primary Completion Date : July 2018

Arm Intervention/treatment
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)

Primary Outcome Measures :
  1. 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 :
  1. 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 ]

Information from the National Library of Medicine

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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): 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

Responsible Party: Alexion Pharmaceuticals Identifier: NCT02324049     History of Changes
Other Study ID Numbers: NGLU-CL02
First Posted: December 24, 2014    Key Record Dates
Last Update Posted: October 11, 2017
Last Verified: October 2017
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