NCT02324049

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

A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 2 Years – 12 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Mucopolysaccharidosis IIIB

Interventions

Type	Name	Description
DRUG	SBC-103

Timeline

• Start date: 2015-01-22
• Primary completion: 2017-10-16
• Completion: 2017-10-16
• First posted: 2014-12-24
• Last updated: 2018-08-21
• Results posted: 2018-07-12

Locations

4 sites across 3 countries: United States, Spain, United Kingdom

Regulatory

• FDA-regulated drug study