Clinical trials in cystic fibrosis

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

Research output: Contribution to journalLiterature reviewpeer-review

41 Citations (Scopus)


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
Pages (from-to)85-99
Number of pages15
JournalJournal of Cystic Fibrosis
Issue number2
Publication statusPublished - Apr 2007


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

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