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

NCT02324049 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: NGLU-CL02
• Study Type: interventional
• Target: 11
• Locations: 3 countries
• Sites: 4

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.

Conditions

Mucopolysaccharidosis IIIB

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)

  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.

  Baseline to Week 142

Study Arms (1)

SBC-103

EXPERIMENTAL

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

Interventions

SBC-103 DRUG

Also known as: recombinant human alpha-N-acetylglucosaminidase

SBC-103

Eligibility Criteria

• Age: 2 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (4)

Unknown Facility

Minneapolis, Minnesota, 55414, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, 15224, United States

Location

Unknown Facility

Barcelona, 08950, Spain

Location

Unknown Facility

Birmingham, B4 6NH, United Kingdom

Location

MeSH Terms

Conditions

Mucopolysaccharidosis III; Mucopolysaccharidoses; Metabolism, Inborn Errors; Metabolic Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Connective Tissue Diseases; Lysosomal Storage Diseases; Mucinoses; Pathologic Processes

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Nutritional and Metabolic Diseases; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

475-230-2596

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2014
• First Posted: December 24, 2014
• Study Start: January 22, 2015
• Primary Completion: October 16, 2017
• Study Completion: October 16, 2017
• Last Updated: August 21, 2018
• Results First Posted: July 12, 2018

Record last verified: 2018-07

Locations

ES (1); GB (1); US (2)