Outcome validation in noses of cystic fibrosis and non-CF subjects

  • Research type

    Research Study

  • Full title

    Outcome validation in noses of subjects with cystic fibrosis and non-CF volunteers



  • Contact name

    Uta Griesenbach

  • Sponsor organisation

    Imperial College London

  • Duration of Study in the UK

    2 years, 4 months, 2 days

  • Research summary

    We have developed a new virus-based gene transfer vector specifically designed to transfer genes into airway lining cells and have recently obtained funding for a first-in-man cystic fibrosis (CF) gene therapy trial. The trial will involve administering the gene therapy agent to the noses of patients with CF and will be covered by a separate NRES application. The nasal lining has a similar cell composition to that of the airways and is frequently used as a convenient surrogate in gene therapy trials. In preparation for the gene therapy trial we will optimise various clinical and laboratory methods for use as safety and efficacy endpoints in the trial. This validation study will take approximately 18 months. We will invite up to 50 adult patients attending the Brompton with CF and 50 subjects without CF and 20 patients with a different condition (called primary ciliary dyskinesia) for comparison. Subjects will attend for up to 3 visits which will coincide with routine clinical appointments. This study will not involve administration of an advanced therapeutic medicinal product.
    Patients will undergo some or all of the tests below. A summary of the visits and sampling/measurements for each participant is available in the flowchart attached to this application.
    (1) Collection of superficial nasal cells using a small brush or a curette to determine the most appropriate method to collect samples suitable for analysis of gene transfer efficiency
    (2) Collection of nasal fluid to quantify inflammatory/immune response markers and develop gene transfer vector shedding assays. Some patients may undergo a nasal washing prior to fluid collection
    (3) Quantification of peak nasal inspiratory flow in CF patient to validate this measurement as a safety outcome
    (4) Collection of blood, urine, saliva to validate potential safety and efficacy endpoints
    (5) collection of sputum to evaluate potential safety and efficacy endpoints

  • REC name

    West Midlands - Solihull Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    25 Sep 2017

  • REC opinion

    Further Information Favourable Opinion

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