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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 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. The trial reports 4 study locations across 3 states.
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).
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03423186
The ClinicalTrials.gov registry entry for NCT03423186 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 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 4 study locations spanning 3 distinct geographic areas - top geographies include 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.
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