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A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2020-07-30

Study results available
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Summary

The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.

Conditions

  • Sly Syndrome
  • MPS VII
  • Mucopolysaccharidosis
  • Mucopolysaccharidosis VII

Interventions

DRUG

UX003

solution for intravenous (IV) infusion

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceuticals, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-11-10
Primary Completion
2019-01-14
Completion
2019-01-14

Countries

  • United States
  • Brazil
  • Mexico
  • Portugal

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 NCT02432144 on ClinicalTrials.gov