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Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

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ClinicalTrials.gov Identifier: NCT02324049
Recruitment Status : Completed
First Posted : December 24, 2014
Results First Posted : July 12, 2018
Last Update Posted : July 12, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
Study to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in participants 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

Detailed Description:
This study was designed as a 3-part study to evaluate the safety and tolerability of IV administration of SBC-103. Participants enrolled in Part A (0.3, 1.0, or 3.0 milligrams [mg] per kilogram [kg] of SBC-103 administered every other week [QOW] for 24 consecutive weeks). Participants who completed Part A were eligible for Part B (an increase to 1.0 or 3.0 mg/kg QOW). Participants who completed Part B were eligible for Part C (5.0 and/or 10.0 mg/kg to continue through Week156; no participants received both 5.0 and 10.0 mg/kg). Due to the early termination of the SBC-103 development program, including this study, all participants withdrew from Part C at the sponsor's decision. As a result of the early termination of this program, this report provides only safety data.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 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
Actual Study Start Date : January 22, 2015
Actual Primary Completion Date : October 16, 2017
Actual Study Completion Date : October 16, 2017


Arm Intervention/treatment
Experimental: SBC-103

Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 mg/kg QOW for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first.

Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C.

Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered IV QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg.

Drug: SBC-103
Other Name: recombinant human alpha-N-acetylglucosaminidase




Primary Outcome Measures :
  1. Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Baseline to Week 142 ]
    TEAEs were defined as any adverse event (AE) that occurred after administration of the first dose of study drug on Day 1 (Part A). A severe AE was defined as an AE that was incapacitating and required medical intervention. TEAEs were summarized cumulatively over the entire study and separately for Part C, data for all severe TEAEs throughout the entire study is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.



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

Key Inclusion Criteria:

  • A participant was 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.
  • Had any internal or non-removable external metal items that presented a safety risk for study assessments that utilized magnetic fields, or any other medical condition or circumstance in which magnetic resonance imaging was 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 ClinicalTrials.gov identifier (NCT number): NCT02324049


Locations
United States, Minnesota
Minneapolis, Minnesota, United States, 55414
United States, Pennsylvania
Pittsburgh, Pennsylvania, United States, 15224
Spain
Barcelona, Spain, 08950
United Kingdom
Birmingham, United Kingdom, B4 6NH
Sponsors and Collaborators
Alexion Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alexion Pharmaceuticals:
Study Protocol  [PDF] January 21, 2016
Statistical Analysis Plan  [PDF] September 1, 2017


Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02324049     History of Changes
Other Study ID Numbers: NGLU-CL02
First Posted: December 24, 2014    Key Record Dates
Results First Posted: July 12, 2018
Last Update Posted: July 12, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases