A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Trial Profile

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Completed
Phase of Trial: Phase III

Latest Information Update: 14 Aug 2017

At a glance

  • Drugs Aceneuramic acid (Primary)
  • Indications Nonaka distal myopathy
  • Focus Registrational; Therapeutic Use
  • Acronyms GNEM
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 09 Aug 2017 Status changed from active, no longer recruiting to completed.
    • 16 Feb 2017 According to an Ultragenyx Pharmaceutical media release, the company plans to submit an NDA based on the results of this trial, if positive.
    • 11 Nov 2016 According to an Ultragenyx Pharmaceutical media release, the company plans to submit an MAA for full approval after data from this study are available in the second half of 2017.
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