Gastroenterological endpoints in drug trials for cystic fibrosis

Abstract

The phenotype of cystic fibrosis includes a wide variety of clinical and biochemical gastrointestinal presentations. These gastrointestinal characteristics of the disease have come under renewed interest as potential outcome measures and clinical endpoints for therapeutic trials in cystic fibrosis. Established gastrointestinal clinical endpoints, like e.g. fecal elastase-1, are already used in trials. Other potential gastrointestinal outcome measures gather more scientific interest for evaluation in future trials. Gastrointestinal outcome measures look particularly relevant and promising for trials in CF patients with normal lung function or therapeutic studies in young children and infants. We review, the currently reported gastrointestinal effects of CFTR modulation therapies and discuss the potential of gastrointestinal outcome measures for therapeutic trials in cystic fibrosis. Pediatr Pulmonol. 2016;51:S18–S22. © 2016 Wiley Periodicals, Inc.