A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

Summary

To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation

Timeline

Start

2016-05

Primary completion

2017-08

Completion

2017-08

Outcome

Missed primary endpoint

Stopped (Enrollment): “Low enrollment.”

registry analysis (superiority test); Placebo (Crossover Part) vs Ivacaftor (Crossover Part); p = 0.2121; t-test, 2 sided NCT02742519 ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ivacaftor	Other / unclassified	—	—

Indications