*Phase 3 open label study of ELX/TEZ/IVA in Cystic Fibrosis Subjects 2 years and older

  • Research type

    Research Study

  • Full title

    A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older



  • Contact name

    Alison Buckland

  • Contact email


  • Sponsor organisation

    Vertex Pharmaceuticals Europe Limited

  • Eudract number


  • Clinicaltrials.gov Identifier


  • Research summary

    Cystic fibrosis (CF) is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Changes in this gene can affect the amount of the protein made from this gene or how well the protein works. When there is not enough protein or it does not work properly, some fluids in the body become sticky which causes problems in the lungs and other organs. This study is being done to learn about the long term safety and effectiveness of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy- which improves CFTR function in patients with certain mutations in their CF genes- in childre who are 2 years and older. This is a Phase 3, 2-part, multicenter, open-label study for subjects who complete parent study VX20-445-111 Part B and meet eligibility criteria. Subjects who complete Part A will have the opportunity to enroll in Part B for an additional 96 weeks.

  • REC name

    North West - Greater Manchester South Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    16 Feb 2022

  • REC opinion

    Further Information Favourable Opinion

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