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VX17-659-105 Open-label Safety and Efficacy of VX-659 in CF

  • Research type

    Research Study

  • Full title

    A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation

  • IRAS ID

    241641

  • Contact name

    Peter Barry

  • Contact email

    peter.barry@mft.nhs.uk

  • Sponsor organisation

    Vertex Pharmaceuticals Incorporated

  • Eudract number

    2017-004134-29

  • Clinicaltrials.gov Identifier

    NCT03447262

  • Duration of Study in the UK

    2 years, 9 months, 16 days

  • 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 and causes problems in the lungs and other organs. VX-659, tezacaftor and ivacaftor are a triple combination of therapies targeted at improving the function of this protein. Early studies have suggested that this triple combination may improve health in some patients with CF. This clinical trial will evaluate how well the triple combination may treat CF in men and women with CF who are 12 years of age or older, and who have certain mutations in their CF genes. In this study, we are testing this triple combination in people with either, one copy of a CF gene with a mutation termed F508del and one copy of a CF gene with a mutation termed a minimal function mutation, or have two copies of the CF gene mutation F508del. The study plans to include participants from VX17-659-102 and VX17-659-103 studies and all participants will receive active treatment (triple combination of VX-659, tezacaftor and ivacaftor). The study duration is 100 weeks, which includes up to 96 weeks of treatment and a follow-up visit after 4 weeks. The study will check if the triple combination can improve lung function and other measures of health in people with cystic fibrosis.

  • REC name

    North East - Tyne & Wear South Research Ethics Committee

  • REC reference

    18/NE/0266

  • Date of REC Opinion

    23 Aug 2018

  • REC opinion

    Favourable Opinion