A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Phase 1

Terminated

• Conditions: Mucopolysaccharidosis III, Type B (MPS IIIB); Sanfilippo B
• Interventions: Drug: SBC-103

• Registration Number: NCT02618512
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study. The NGLU-CL01 study was a non-interventional study that evaluated structural brain abnormalities and blood brain barrier (BBB) integrity by magnetic resonance imaging (MRI) and cerebrospinal fluid/serum albumin index.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-10-15
• Study First Submitted: 2015-10-21
• Study First Submitted QC: 2015-11-25
• Study First Posted: 2015-12-01
• Primary Completion: 2017-08-18
• Study Completion: 2017-08-18
• Results First Submitted: 2018-02-16
• Status Verified: 2018-03
• Results First Submitted QC: 2018-03-19
• Last Update Submitted: 2018-03-19
• Results First Posted: 2018-04-17
• Last Update Posted: 2018-04-17

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
SBC-103	SBC-103	Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days.

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability of SBC-103	Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.	The planned primary endpoint of this study was safety and tolerability of SBC-103 in patients with MPS IIIB, as measured by Number of participants with treatment-emergent adverse events, including serious adverse events; infusion-associated reactions; incidence of antidrug antibodies, clinical laboratory tests, cerebrospinal fluid findings, vital signs, and prior and concomitant medications