CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 8 enrolled
Drug / intervention
UX003drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT02418455
NCT02418455Phase 2Completed
CompareCT.gov

An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old

Ultragenyx Pharmaceutical Inc·interventional·Posted Apr 16, 2015·Updated Oct 30, 2019

In Brief

A Phase 2 clinical trial evaluating UX003 for Sly Syndrome and 3 related conditions. Completed, enrolled 8 participants across 5 sites in 3 countries.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
CountriesPortugal, Spain, United States
Collaborators--

Timeline

Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedApr 16, 2015
Enrollment StartJul 21, 2015
Primary CompletionMar 26, 2019
TodayJul 2, 2026
Enrollment to primary: 3.7 yearsPosted 11.2 years ago

Interventions

UX003drug

solution for intravenous infusion