Phase 2
Completed N=11
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Mucopolysaccharidosis IIIB
Source: ClinicalTrials.gov NCT02324049 ↗
Enrolled (actual)
11
Serious AEs
27.3%
Results posted
Jul 2018
Primary outcomePrimary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) |
1; 0; 1 | — |
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT02324049). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.