CompletedPhase 1/2

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

United States, Spain, United Kingdom11 participantsStarted 2015-01-22

Plain-language 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.

Who can participate

Age range2 Years – 12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)

Timeframe: Baseline to Week 142

Trial details

NCT IDNCT02324049

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-10-16

Results submitted2018-06-12

View on ClinicalTrials.gov More Mucopolysaccharidosis IIIB trials