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

• Status: Completed
• Trial end date: 2017-10-16
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

Criteria

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.