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 (CL01-T)

Alexion Pharmaceuticals

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Sanfilippo B; Mucopolysaccharidosis III, Type B (MPS IIIB)

Treatments: Drug: SBC-103

Study type: Interventional

Funder types: Industry

Identifiers

NCT02618512

2015-001983-20 (EudraCT Number)

NGLU-CL01-T

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: All

Ages: 5+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Participants from NGU-CL01 study were enrolled into the NGLU-CL01-T study. Subjects who met all of the inclusion criteria and none of the exclusion criteria were eligible to participate in this study.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

SBC-103

Experimental group

Description:

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.

Treatment:

Drug: SBC-103