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NCT02324049 · ClinicalTrials.gov registry record · Phase 1
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
A Phase 1 study of Mucopolysaccharidosis IIIB, sponsored by Alexion Pharmaceuticals.
- Completed
- Registry status
- Phase 1
- Development phase
- 11
- Enrollment target
- 4
- Study locations
NCT02324049: Completed Phase 1 study of Mucopolysaccharidosis IIIB, sponsored by Alexion Pharmaceuticals.
NCT02324049 is a Phase 1 study of Mucopolysaccharidosis IIIB that has completed, run by Alexion Pharmaceuticals. The registered enrollment target is 11 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (82% lower). The trial reports 4 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02324049, a Phase 1 study of Mucopolysaccharidosis IIIB, has completed, sponsored by Alexion Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 11 participants
- Enrollment target
- 4
- Study locations
Study 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.
Primary Outcome
TEAEs were defined as any adverse event (AE) that occurred after administration of the first dose of study drug on Day 1 (Part A). A severe AE was defined as an AE that was incapacitating and required medical intervention. TEAEs were summarized cumulatively over the entire study and separately for Part C, data for all severe TEAEs throughout the entire study is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Conditions Studied
Interventions
- DRUG SBC-103
Study Locations (4)
Other
- - Barcelona
- - Birmingham
Minnesota
- - Minneapolis
Pennsylvania
- - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2015-01-22 |
| Est. Completion | 2017-10-16 |
| Phase | Phase 1 |
What the finished NCT02324049 record still lists
NCT02324049 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 11 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (82% lower).
The record links to 1 condition, with Mucopolysaccharidosis IIIB appearing as the primary indexed condition, and to 1 intervention - of which SBC-103 is the first listed.
NCT02324049 reports a single indexed study location in Other, Minnesota, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT02324049 about?
NCT02324049 is a clinical study titled "Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)". 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.
What is the current status of trial NCT02324049?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 11 participants. The study started on 2015-01-22. Estimated completion is 2017-10-16.
What conditions does trial NCT02324049 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis IIIB.
What interventions are being tested in trial NCT02324049?
The interventions under investigation include: SBC-103 (DRUG).
Who is sponsoring clinical trial NCT02324049?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02324049 being conducted?
This trial has 4 study locations across Minnesota, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02324049's enrollment target sits among peer trials
11 1911th of 2000 higher than 88 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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