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: 21 Sep 2017

At a glance

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

    • 22 Aug 2017 According to an Ultragenyx Pharmaceutical media release, based on results of this trial Ultragenyx plans to discontinue further clinical development of Ace-ER.
    • 22 Aug 2017 Results published in an Ultragenyx Pharmaceutical media release.
    • 22 Aug 2017 Primary endpoint (Upper Extremity Muscle Strength) has not been met.
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