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VX17-661-116 Open-label safety and efficacy of TEZ/IVA 6 yrs and above

  • Research type

    Research Study

  • Full title

    A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation

  • IRAS ID

    241643

  • Contact name

    Timothy Lee

  • Contact email

    tim.lee1@nhs.net

  • Sponsor organisation

    Vertex Pharmaceuticals (Europe) Ltd

  • Eudract number

    2017-002968-40

  • Clinicaltrials.gov Identifier

    NCT03537651

  • Duration of Study in the UK

    2 years, 4 months, 1 days

  • Research summary

    Cystic Fibrosis (CF) is a genetic disease caused by mutations in the gene encoding a cell transport protein (ion channel). This ion channel transports chloride ions in and out of cells in multiple organ systems. When ions are not transported correctly the salt and water balance in cells and tissues is not controlled, leading to the production of sticky mucus in the lungs, airways and intestines.

    This clinical study is designed to evaluate the long-term safety and efficacy of tezacaftor/ivacaftor in subjects with CF aged 6 years or more, who are homozygous (two copies) or heterozygous (one copy) for F508del along with a second (minimal function) CF mutation. Tezacaftor (TEZ) and ivacaftor (IVA)both address the underlying cause of CF.

    The study is designed to evaluate the safety and efficacy of long-term TEZ/IVA treatment in paediatric subjects aged 6 years and older. Subjects who complete treatment in the parent study VX16-661-115 will be offered the opportunity to enrol into VX17-661-116. All participants will receive active study drug for up to 96 weeks.

  • REC name

    East of Scotland Research Ethics Service REC 2

  • REC reference

    18/ES/0054

  • Date of REC Opinion

    28 Jun 2018

  • REC opinion

    Further Information Favourable Opinion