A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Trial Profile

A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Completed
Phase of Trial: Phase III

Latest Information Update: 15 Jan 2018

At a glance

  • Drugs Ivacaftor/lumacaftor (Primary)
  • Indications Cystic fibrosis
  • Focus Adverse reactions; Pharmacokinetics; Registrational
  • Sponsors Vertex Pharmaceuticals
  • Most Recent Events

    • 25 Oct 2017 According to a Vertex Pharmaceuticals media release, the company expects to submit a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) line extension to the European Medicines Agency (EMA) in the first quarter of 2018 based on results from this study.
    • 25 Oct 2017 According to a Vertex Pharmaceuticals media release, the company has announced results from this trial.
    • 12 Sep 2017 Status changed from active, no longer recruiting to completed.
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