DESIGN AND POWERING OF CYSTIC FIBROSIS CLINICAL TRIALS USING PULMONARY EXACERBATION AS AN EFFICACY ENDPOINT

摘要

BACKGROUNDReduction in pulmonary exacerbations is an important efficacy endpoint for CF clinical studies. Powering exacerbation endpoints requires estimation of the future exacerbation incidence in CF study populations, but rates differ across the population.