An Open-label Phase 2 Extension Study to Evaluate the Long Term Safety and Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Trial Profile

An Open-label Phase 2 Extension Study to Evaluate the Long Term Safety and Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 14 Apr 2016

At a glance

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

    • 08 Apr 2016 Planned primary completion date changed from 1 Jun 2016 to 1 Feb 2017.
    • 10 Nov 2014 Two-year results published in Ultragenyx Pharmaceutical media release.
    • 12 May 2014 Ultragenyx continues to treat patients in this extension study evaluating an increased daily dosage of sialic acid based on the dose dependent response observed at weeks 24 and 48 of the original phase II study.
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