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Pediatr Pulmonol. 2015 Mar;50(3):302-315. doi: 10.1002/ppul.23146. Epub 2014 Dec 30.

Novel outcome measures for clinical trials in cystic fibrosis.

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Department of Pediatric Pulmonology and Allergology, Department of Radiology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
Department for Diagnostic and Interventional Radiology, Hufeland Klinikum, Bad Langensalza, Germany.
Department of Anesthesia, Critical Care and Pain Medicine, Massachusetts General Hospital, Boston, Massachusetts.
Department of Pediatrics, Division of Respiratory Medicine, Hospital for Sick Children, University of Toronto, Toronto, Ontario.
Department of Medicine, University of North Carolina, Chapel Hill, North Carolina.
Department of Pediatrics, James Whitcomb Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana.
Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama.
Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Denver, Colorado.
Vertex Pharmaceuticals Europe, Abingdon, UK.
Vertex Pharmaceuticals, Boston, Massachusetts.


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. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.


CFTR activity; cystic fibrosis; endpoints; imaging; outcome measures; sputum biomarkers

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