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

Trial Profile

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

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 10 Mar 2017

At a glance

  • Drugs Vestronidase alfa (Primary)
  • Indications Mucopolysaccharidosis VII
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 06 Mar 2017 Planned number of patients changed from 7 to 15.
    • 06 Mar 2017 Status changed from recruiting to active, no longer recruiting.
    • 05 Aug 2015 According to an Ultragenyx Pharmaceuticals media release, first patient has been dosed in this trial. Interim data from this study are expected by the end of 2016. This study is also intended to enroll patients with non-immune hydrops fetalis.
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top