Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review.

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Pediatric Pulmonology
Department of Pediatrics

Tiddens, H., Puderbach, M., Venegas, J. G., Ratjen, F., Donaldson, S., Davis, S., … Waltz, D. (2015). Novel outcome measures for clinical trials in cystic fibrosis. Pediatric Pulmonology, 50(3), 302–315. doi:10.1002/ppul.23146