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

Alexion Pharmaceuticals, Inc.11 enrolled

Overview

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.

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

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Mucopolysaccharidosis IIIB

Interventions

SBC-103DRUG

Eligibility

Sex: ALLMin age: 2 YearsMax age: 12 Years

Medical Language ↔ Plain English

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.

Locations (4)

Unnamed facility

Minneapolis, Minnesota, United States

Unnamed facility

Pittsburgh, Pennsylvania, United States

Unnamed facility

Barcelona, Spain

Unnamed facility

Birmingham, United Kingdom

Outcomes

Primary Outcomes

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.

Time frame: Baseline to Week 142

Data from ClinicalTrials.gov

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