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

Mucopolysaccharidosis Clinical Trial

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

Status Completed

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02418455
Study type	Interventional
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Completed
Phase	Phase 2
Start date	July 21, 2015
Completion date	March 26, 2019

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT01695161` - Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.	N/A
Recruiting	`NCT01238328` - Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis	Phase 2/Phase 3
Completed	`NCT02432144` - A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)	Phase 3
Terminated	`NCT00654433` - ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases	Phase 3
Completed	`NCT05006222` - The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis	N/A
Completed	`NCT02230566` - A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)	Phase 3
Completed	`NCT01043640` - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders	Phase 2