VX15-770-126 Cystic Fibrosis

  • Research type

    Research Study

  • Full title

    A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation



  • Contact name

    Antoinette Paone

  • Contact email


  • Sponsor organisation

    Vertex Pharmaceuticals Incorporated

  • Eudract number


  • Duration of Study in the UK

    5 years, 0 months, 27 days

  • Research summary

    This is a phase 3, 2 part, open-label (no placebo or ‘dummy drug’) study to assess the safety and effects of long-term use of the study drug Ivacaftor in young children with cystic fibrosis (CF). CF is a rare genetic disease, that affects the lungs, the digestive system and other organs.

    Ivacaftor (Kalydeco®) is a potentiator (i.e. improves the function) of the faulty protein, CFTR. It was the first therapy to target the underlying cause of CF and is licensed for children aged 2 years and above with a specific group of CFTR gene mutations.

    This study of children aged less than 2 years is designed to learn more about the safety and effects of long term Ivacaftor treatment. The study is split into 2 arms; one arm the participants will receive ivacaftor treatment for 104 weeks with a follow up period of 24 weeks and an observational arm with follow-up for 104 weeks (receiving no ivacaftor).

    There will be approximately 75 participants enrolled into the study globally

  • REC name

    London - Chelsea Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    20 Jul 2017

  • REC opinion

    Favourable Opinion

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