A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment

Trial Profile

A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment

Recruiting
Phase of Trial: Phase II

Latest Information Update: 13 Aug 2017

At a glance

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

    • 10 Jun 2017 Biomarkers information updated
    • 14 May 2017 Planned number of patients changed from 30 to 45.
    • 12 Apr 2017 Planned primary completion date changed from 1 Aug 2017 to 1 Aug 2018.
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