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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

NCT02418455 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2019-10-30

Study results available
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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.

Conditions

  • Sly Syndrome
  • MPS VII
  • Mucopolysaccharidosis
  • Mucopolysaccharidosis VII

Interventions

DRUG

UX003

solution for intravenous infusion

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Day
Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-07-21
Primary Completion
2019-03-26
Completion
2019-03-26

Countries

  • United States
  • Portugal
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02418455 on ClinicalTrials.gov