A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

Summary

The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation, and being treated with Orkambi

Timeline

Start

2016-02

Primary completion

2017-09

Completion

2017-09

Outcome

Missed primary endpoint

registry analysis (superiority test); VX-371 + Hypertonic Saline vs Hypertonic Saline; p = 0.8173; Least Square (LS) mean difference 0.2 (95% CI -1.4 to 1.8) NCT02709109 ↗

Drugs

Indications