Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Trial Profile

Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 18 Sep 2017

At a glance

  • Drugs Aceneuramic acid (Primary)
  • Indications Nonaka distal myopathy
  • Focus Adverse reactions
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 14 Sep 2017 Status changed from recruiting to active, no longer recruiting.
    • 12 Apr 2017 Planned primary completion date changed from 1 Jan 2020 to 1 Apr 2020.
    • 01 Nov 2016 Planned number of patients changed from 110 to 165.
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