Novel outcome measures for clinical trials in cystic fibrosis

Abstract

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.

Keywords: CFTR activity; cystic fibrosis; endpoints; imaging; outcome measures; sputum biomarkers.

Figure 1

Stages of disease progression and pathologic changes that occur in the airways of patients with CF as they age, along with possible treatment approaches. Reprinted with permission of the American Thoracic Society. Copyright © 2014 American Thoracic Society. Ramsey BW. 2007. Use of lung imaging studies as outcome measures for development of new therapies in cystic fibrosis. Proc Am Thorac Soc;4(4):359–63. Official Journal of the American Thoracic Society.