Clinical Trial: An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age

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

Official Title:

Brief Summary: UX003-CL203 is an open-label, multi-center, Phase 2 study to assess the safety and efficacy of UX003 in pediatric MPS 7 subjects.

Detailed Summary:
Sponsor: Ultragenyx Pharmaceutical Inc

Current Primary Outcome:

• Efficacy of UX003 (percent reduction of uGAG excretion) [ Time Frame: 48 weeks ]
  Determined by the percent reduction of uGAG excretion after 48 weeks of treatment relative to the pre-treatment baseline.
• Safety and tolerability of UX003 (severity, frequency, and number of Adverse Events and Serious Adverse Events) [ Time Frame: 48 weeks ]
  Determined by the severity, frequency, and number of AEs and SAEs.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Efficacy of UX003 (changes in growth velocity) [ Time Frame: 48 weeks ]
  Determined by changes in growth velocity after 48 weeks of treatment relative to pre-treatment baseline and published normative data for age and gender.
• Efficacy of UX003 (volume of the liver and spleen) [ Time Frame: 48 weeks ]
  Determined by the volume of the liver and spleen after 48 weeks of treatment relative to pre-treatment baseline and normal values for age and gender.

Original Secondary Outcome: Same as current

Information By: Ultragenyx Pharmaceutical Inc

Dates:
Date Received: April 12, 2015
Date Started: July 2015
Date Completion:
Last Updated: March 6, 2017
Last Verified: March 2017