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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 is a Phase 3 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 12 participants.

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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).

Interventions

  • OTHER Placebo
  • DRUG UX003

Trial Details

FieldValue
Enrollment Target 12 participants
Start Date 2014-12
Est. Completion 2016-05
Phase Phase 3

Sponsor

Ultragenyx Pharmaceutical

52 total trials

What the Registry Record Tells You About NCT02230566

The ClinicalTrials.gov registry entry for NCT02230566 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 52 total studies on file at ClinicalTrials.gov.

The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.

NCT02230566 reports 0 study locations.

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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.