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NCT02418455 · ClinicalTrials.gov registry record · Phase 2
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
A Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 8
- Enrollment target
NCT02418455: Completed Phase 2 study, sponsored by Ultragenyx Pharmaceutical.
NCT02418455 is a Phase 2 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 8 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (94% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02418455, a Phase 2 study, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 8 participants
- Enrollment target
Study Summary
The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans (uGAG) excretion.
Primary Outcome
Liquid chromatography-mass spectrometry/mass spectrometry-dermatan sulfate (LS-MS/MS-DS) method. For the participant previously treated with UX003 under an eIND, percent change from initial baseline was used.
Interventions
- DRUG UX003
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2015-07-21 |
| Est. Completion | 2019-03-26 |
| Phase | Phase 2 |
What the finished NCT02418455 record still lists
NCT02418455 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (94% lower).
The record links to 0 conditions, and to 1 intervention - of which UX003 is the first listed.
NCT02418455 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02418455 about?
NCT02418455 is a clinical study titled "Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age". The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans (uGAG) excretion.
What is the current status of trial NCT02418455?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 8 participants. The study started on 2015-07-21. Estimated completion is 2019-03-26.
What interventions are being tested in trial NCT02418455?
The interventions under investigation include: UX003 (DRUG).
Who is sponsoring clinical trial NCT02418455?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02418455's enrollment target sits among peer trials
8 1990th of 2000 higher than 5 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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