Adherence to LUM and IVA in CF

  • Research type

    Research Study

  • Full title

    A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallel-group, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del-CFTR Mutation

  • IRAS ID

    214042

  • Contact name

    Gordon MacGregor

  • Contact email

    gordon.macgregor@ggc.scot.nhs.uk

  • Sponsor organisation

    Vertex Pharmaceuticals

  • Clinicaltrials.gov Identifier

    NCT02823470

  • Duration of Study in the UK

    1 years, 10 months, 17 days

  • Research summary

    This is a phase IV, Open-label, randomised study to monitor adherence to Lumacaftor(LUM)/Ivacaftor(IVA) in Cystic Fibrosis patients.

    The purpose of the study is to learn more about the use of Smart Adherence Technology (smart pill bottles) in patients with Cystic Fibrosis while taking LUM/IVA combination treatment.

    About 75 people will participate in the study. Eligible patients will receive LUM and IVA, which has been approved for the treatment of CF by the MHRA. Patients will be randomly assigned to receive either an activated smart pill bottle or a control smart pill bottle, which will be de-activated. Patients will remain in the study for 48 weeks.

    The smart pill bottles offer a method for CF patients and physicians to objectively monitor medication compliance and to make long-term adherence manageable. Studies have shown that 30-50% of medicines are not taken as recommended and adherence tends to decline after the first 6 months of treatment.

    A number of safety assessments and procedures will be performed during the study including physical examinations, blood sample assessments, patient compliance questionnaires and pregnancy tests (if applicable).

    The study will be run at approximately 8 to 10 centres in the UK and is sponsored by Vertex Pharmaceuticals Limited.

  • REC name

    West of Scotland REC 5

  • REC reference

    16/WS/0257

  • Date of REC Opinion

    20 Dec 2016

  • REC opinion

    Unfavourable Opinion