Clinical Trial: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7

Brief Summary: UX003-CL202 is an open-label, multi-center extension study to assess long-term safety and efficacy of UX003 treatment in patients with MPS 7.

Detailed Summary:

Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome) is an ultra-rare (< 100 cases currently identified worldwide), chronically debilitating and life threatening lysosomal storage disease. It is characterized by a deficiency of the lysosomal enzyme beta-glucuronidase (GUS), required for degradation of the glycosaminoglycans (GAGs): dermatan sulfate (DS), chondroitin-6-sulfate (CS) and heparan sulfate (HS). The GUS deficiency results in lysosomal accumulation of GAGs in multiple tissues and organs throughout the body and numerous clinical signs and symptoms as a result of tissue damage and organ dysfunction. There are currently no approved treatments for MPS 7.

UX003 (recombinant human beta glucuronidase, rhGUS) is intended as a long-term enzyme replacement therapy (ERT) for the treatment of MPS 7 via intravenous (IV) administration. Ultragenyx is conducting this treatment and extension study to assess the long-term safety and efficacy of UX003 treatment in subjects with MPS 7. Subjects with MPS 7 who are UX003 treatment-naïve or have been previously enrolled and treated with UX003 in other clinical studies or programs are eligible for enrollment.

The study will continue for up to 144 weeks or until one of the following occurs: the subject withdraws consent and discontinues from the study, the subject is discontinued from the study at the discretion of the Investigator or Ultragenyx, or the study is terminated.


Sponsor: Ultragenyx Pharmaceutical Inc

Current Primary Outcome: Safety of UX003 determined by the incidence and frequency of adverse events (AEs), treatment related AEs, and Serious Adverse Events (SAEs) [ Time Frame: 144 weeks ]

Determined by the incidence and frequency of adverse events (AEs), treatment related AEs, and Serious Adverse Events (SAEs).


Original Primary Outcome: Same as current

Current Secondary Outcome: Efficacy of UX003 determined by the percent reduction and change from baseline of uGAG excretion [ Time Frame: 144 weeks ]

Determined by the percent reduction and change from baseline of uGAG excretion over 144 weeks of treatment.


Original Secondary Outcome: Same as current

Information By: Ultragenyx Pharmaceutical Inc

Dates:
Date Received: April 22, 2015
Date Started: October 2015
Date Completion: February 2019
Last Updated: April 18, 2017
Last Verified: April 2017