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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 is a Phase 1 study that has completed, run by Swedish Orphan Biovitrum. The registered enrollment target is 6 participants.
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).
Interventions
- DRUG SOBI003
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2019-01-19 |
| Est. Completion | 2021-05-07 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03811028
The ClinicalTrials.gov registry entry for NCT03811028 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Swedish Orphan Biovitrum, which has 23 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which SOBI003 is the first listed.
NCT03811028 reports 0 study locations.
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.
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