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Phase 3 Open-label Study of VX-445/TEZ/IVA in subjects 6 years & older

  • Research type

    Research Study

  • Full title

    A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older

  • IRAS ID

    274499

  • Contact name

    Jane Davies

  • Contact email

    j.c.davies@imperial.ac.uk

  • Sponsor organisation

    Vertex Pharmaceuticals Incorporated

  • Eudract number

    2019-001827-11

  • Duration of Study in the UK

    1 years, 6 months, 0 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. This study will help researchers learn if the study drugs VX-445/TEZ/IVA is safe and effective in the tested doses. VX- 445, tezacaftor and ivacaftor are a triple combination of therapies targeted at improving the function of this protein. There is a strong rationale for preventing the progress of the disease by treating patients earlier in life. Currently Vertex VX-445/TEZ/IVA therapy in Phase 3 studies in adult and young CF subjects with 1 or 2 copies of the F508del mutation, namely those with F/MF and F/F genotypes. Furthermore this triple combination therapy has been approved by FDA for people with CF aged 12 years and older who have at least one copy of the F508del mutation, regardless of their second mutation. Given the clinical benefit seen in previous studies of adults with CF, this study is designed to obtain Long-term Safety and Efficacy of the study drug in the pediatric population 6-11 years of age which is the open label extension study of an already approved study (VX18-445-106)
    Approximately 56 subjects are planned for enrollment in this study. Participants will be involved in this study for approximately 100 weeks. This will include A study treatment period that will last up to 96 weeks and A follow-up period that will last up to 4 weeks.

  • REC name

    North West - Greater Manchester South Research Ethics Committee

  • REC reference

    20/NW/0004

  • Date of REC Opinion

    24 Apr 2020

  • REC opinion

    Further Information Favourable Opinion