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NCT03811028 · ClinicalTrials.gov registry record · Phase 1
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
A Phase 1 study, sponsored by Swedish Orphan Biovitrum.
- Completed
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
NCT03811028: Completed Phase 1 study, sponsored by Swedish Orphan Biovitrum.
NCT03811028 is a Phase 1 study 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). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03811028, a Phase 1 study, has completed, sponsored by Swedish Orphan Biovitrum.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
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 aim of the present study is to assess the safety, tolerability and efficacy of long-term SOBI003 treatment. SOBI003 is 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 week 25 up to week 104
Interventions
- DRUG SOBI003
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2019-01-19 |
| Est. Completion | 2021-05-07 |
| Phase | Phase 1 |
What the finished NCT03811028 record still lists
NCT03811028 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 0 conditions, and to 1 intervention - of which SOBI003 is the first listed.
NCT03811028 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03811028 about?
NCT03811028 is a clinical study titled "A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment 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 NCT03811028?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2019-01-19. Estimated completion is 2021-05-07.
What interventions are being tested in trial NCT03811028?
The interventions under investigation include: SOBI003 (DRUG).
Who is sponsoring clinical trial NCT03811028?
This trial is sponsored by Swedish Orphan Biovitrum, which has 23 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03811028'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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