Novel Outcome Measures for Clinical Trials in Cystic Fibrosis

Overview

Journal Pediatr Pulmonol

Specialties Pediatrics
Pulmonary Medicine

Date 2015 Feb 3

PMID 25641878

Citations 13

Authors

Harm A W M Tiddens

Michael Puderbach

Jose G Venegas

Felix Ratjen

Scott H Donaldson

Stephanie D Davis

Steven M Rowe

Scott D Sagel

Mark Higgins

David A Waltz

Affiliations

Soon will be listed here.

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.

Citing Articles

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