VX20-121-102 - Phase 3, Randomized, Double-blind, Controlled Study

  • Research type

    Research Study

  • Full title

    A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)



  • Contact name

    Stacey Lindmark

  • Contact email


  • Sponsor organisation

    Vertex Pharmaceuticals Incorporated

  • Eudract number


  • 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). 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 and causes problems in the lungs and other organs.
    This study will evaluate the effectiveness and and safety of VX-121/TEZ/D-IVA in CF subjects who are homozygous (having two identical alleles of a particular gene or genes) for F508del (F/F), heterozygous (having two different alleles of a particular gene or genes) for F508del and a gating (F/G) or a (F/RF) mutation, or have at least 1 triple combination responsive CFTR mutation
    The total study duration is approximately 64 weeks (4 weeks for the Screening Period, 4 weeks for the Run-in Period, 52 weeks for the Treatment Period, and 4 weeks for the Safety Follow-up Period).

  • REC name

    North West - Haydock Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    24 Nov 2021

  • REC opinion

    Further Information Favourable Opinion

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