A Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Single and Repeat Doses of Sialic Acid Extended Release (SA-ER) Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Trial Profile

A Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Single and Repeat Doses of Sialic Acid Extended Release (SA-ER) Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Completed
Phase of Trial: Phase I

Latest Information Update: 02 Oct 2015

At a glance

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

    • 02 Oct 2015 According to Ultragenyx media release, the company has filed a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for aceneuramic acid prolonged release (AceER; UX001) tablets intended for patients with GNE Myopathy. The submission has been accepted for review and the company expects a decision from the European Commission in the second half of 2016.
    • 04 Dec 2014 New trial record
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