Clinical trials in cystic fibrosis

    Research output: Contribution to journalLiterature review

    Published
    • Gerd Döring
    • J. Stuart Elborn
    • Marie Johannesson
    • Hugo de Jonge
    • Matthias Griese
    • Alan Smyth
    • Harry Heijerman

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    In patients with cystic fibrosis (CF), clinical trials are of paramount importance. Here, the current status of drug development in CF is discussed and future directions highlighted. Methods for pre-clinical testing of drugs with potential activity in CF patients including relevant animal models are described. Study design options for phase II and phase III studies involving CF patients are provided, including required patient numbers, safety issues and surrogate end point parameters for drugs, tested for different disease manifestations. Finally, regulatory issues for licensing new therapies for CF patients are discussed, including new directives of the European Union and the structure of a European clinical trial network for clinical studies involving CF patients is proposed.
    Original languageEnglish
    Number of pages15
    Pages (from-to)85-99
    JournalJournal of Cystic Fibrosis
    Journal publication dateApr 2007
    Issue number2
    Volume6
    DOIs
    Publication statusPublished - Apr 2007

      Research areas

    • drug development, pre-clinical drug testing, animal models, surrogate end points, safety issues, drug licensing , european clinical trial network for CF, Consesnsus study Group

    ID: 3464096