An Open-label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Trial Profile

An Open-label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Completed
Phase of Trial: Phase II

Latest Information Update: 11 Sep 2018

At a glance

  • Drugs Triheptanoin (Primary)
  • Indications Lipid metabolism disorders
  • Focus Therapeutic Use
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 11 Sep 2018 According to an Ultragenyx Pharmaceutical media release, in the EU, Ultragenyx will discuss the data submitted to the FDA with the European Medicines Agency (EMA) and expects to have an update in the second half of 2018.
    • 11 Sep 2018 According to an Ultragenyx Pharmaceutical media release, the U.S. FDA has accepted Ultragenyxs most recent proposal to submit a NDA for UX007 for the treatment of long-chain fatty acid oxidation disorders based on existing data.The data submitted to the FDA for evaluation included the recently published results from this trial along with results of the retrospective study.In addition, these data were supported by results of a randomized controlled investigator study (NCT01379625).
    • 30 Nov 2016 Results of 78-week published in an Ultragenyx Pharmaceutical media release.
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