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A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

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Trial Profile

A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 09 Jan 2023

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At a glance

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

Most Recent Events

  • 28 Oct 2022 study protocol amended as primary endpoint (Pk) changed from AUC to Observed Plasma Concentrations, one more end-point (Observed Pre-dose Plasma Concentration) has been added and time frame of endpoint (Ar) has been changed to [From Day 1 up to Week 26].
  • 02 Sep 2022 According to a Vertex Pharmaceuticals media release, based on this trial the U.S. Food and Drug Administration (FDA) has approved expanded use of ORKAMBI (lumacaftor/ivacaftor) to include children with cystic fibrosis (CF) ages 12 to <24 months who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
  • 30 Jun 2022 Results published in the American Journal of Respiratory and Critical Care Medicine

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