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NCT02230566 · ClinicalTrials.gov registry record · Phase 3
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
A Phase 3 study, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 12
- Enrollment target
NCT02230566: Completed Phase 3 study, sponsored by Ultragenyx Pharmaceutical.
NCT02230566 is a Phase 3 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 12 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02230566, a Phase 3 study, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 12 participants
- Enrollment target
Study Summary
The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003. The Blind Start is a novel design whereby participants will be randomized to 1 of 4 groups, each representing a different treatment sequence, and will cross over to UX003 at different pre-defined time points in a blinded manner. All groups will receive a minimum of 24 weeks treatment with 4 mg/kg UX003 every other week (QOW).
Primary Outcome
Baseline was defined as the average of all assessments prior to or on the date of cross-over to active treatment with UX003. (This provides the valid assessment, as placebo effect was subtracted from the treatment effect estimate providing a more conservative assessment of the true treatment effect.) Percent change from baseline in uGAG DS was analyzed by generalized estimating equation (GEE) modeling based on observed data. The GEE model included included baseline value, and the UX003 treatment
Interventions
- OTHER Placebo
- DRUG UX003
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2014-12 |
| Est. Completion | 2016-05 |
| Phase | Phase 3 |
What the finished NCT02230566 record still lists
NCT02230566 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT02230566 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02230566 about?
NCT02230566 is a clinical study titled "A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)". The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003. The Blind Start is a novel design whereby participants will be randomized to 1 of 4 groups, each representing a differen...
What is the current status of trial NCT02230566?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 12 participants. The study started on 2014-12. Estimated completion is 2016-05.
What interventions are being tested in trial NCT02230566?
The interventions under investigation include: Placebo (OTHER), UX003 (DRUG).
Who is sponsoring clinical trial NCT02230566?
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 NCT02230566's enrollment target sits among peer trials
12 1984th of 2000 higher than 12 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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