A Randomized, Double-blind, Placebo-controlled, Parallel-group, Study to Assess the Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome.

Trial Profile

A Randomized, Double-blind, Placebo-controlled, Parallel-group, Study to Assess the Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome.

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 20 Jul 2017

At a glance

  • Drugs Triheptanoin (Primary)
  • Indications De Vivo disease
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors Ultragenyx Pharmaceutical
  • Most Recent Events

    • 20 Jul 2017 The study has been completed in Spain.
    • 22 Mar 2017 Topline results published in an Ultragenyx Pharmaceutical media release.
    • 22 Mar 2017 Primary endpoint (Efficacy of UX007 compared to placebo as measured by reduction from randomization to week 8 in frequency of seizures. Observable generalized and partial-onset seizures measured by diary) has not been met, according to an Ultragenyx Pharmaceutical media release.
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