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NCT03423186 · ClinicalTrials.gov registry record · Phase 1
A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
A Phase 1 study of Sanfilippo Syndrome Type A (MPS IIIA), sponsored by Swedish Orphan Biovitrum.
- Completed
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 4
- Study locations
NCT03423186: Completed Phase 1 study of Sanfilippo Syndrome Type A (MPS IIIA), sponsored by Swedish Orphan Biovitrum.
NCT03423186 is a Phase 1 study of Sanfilippo Syndrome Type A (MPS IIIA) that has completed, run by Swedish Orphan Biovitrum. The registered enrollment target is 6 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower). The trial reports 4 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03423186, a Phase 1 study of Sanfilippo Syndrome Type A (MPS IIIA), has completed, sponsored by Swedish Orphan Biovitrum.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 4
- Study locations
Study Summary
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG) heparan sulfate (HS). The natural course of MPS IIIA is characterized by devastating neurodegeneration with initially mild somatic involvement. The aims of the present study is to assess the dose related safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SOBI003, a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
Primary Outcome
Number of adverse events, by type and severity, from start of infusion up to 24 weeks
Conditions Studied
Interventions
- DRUG SOBI003
Study Locations (4)
Other
- University Medical Center Hamburg-Eppendorf - Hamburg
- Gazi University Hospital - Ankara
California
- Childrens's Hospital and Research Center - Oakland
North Carolina
- University of North Carolina Hospitals - Chapel Hill
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2018-06-19 |
| Est. Completion | 2019-10-25 |
| Phase | Phase 1 |
What the finished NCT03423186 record still lists
NCT03423186 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower).
The record links to 1 condition, with Sanfilippo Syndrome Type A (MPS IIIA) appearing as the primary indexed condition, and to 1 intervention - of which SOBI003 is the first listed.
NCT03423186 reports a single indexed study location in Other, California, North Carolina.
Frequently Asked Questions
What is clinical trial NCT03423186 about?
NCT03423186 is a clinical study titled "A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients". MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG) heparan sulfate (HS). The natural course of MPS IIIA is characterized by d...
What is the current status of trial NCT03423186?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2018-06-19. Estimated completion is 2019-10-25.
What conditions does trial NCT03423186 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome Type A (MPS IIIA).
What interventions are being tested in trial NCT03423186?
The interventions under investigation include: SOBI003 (DRUG).
Who is sponsoring clinical trial NCT03423186?
This trial is sponsored by Swedish Orphan Biovitrum, which has 23 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03423186 being conducted?
This trial has 4 study locations across California, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03423186's enrollment target sits among peer trials
6 1974th of 2000 higher than 12 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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