Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR.

ABSTRACT:

Rationale

In a prior study, lumacaftor/ivacaftor treatment (≤28 d) in patients with cystic fibrosis (CF) heterozygous for F508del-CFTR did not improve lung function.

Objectives

To evaluate an optimized lumacaftor/ivacaftor dosing regimen with a longer duration in a cohort of patients heterozygous for F508del-CFTR.

Methods

Patients aged 18 years or older with a confirmed CF diagnosis and percent predicted FEV₁ (ppFEV₁) of 40 to 90 were randomized to lumacaftor/ivacaftor (400 mg/250 mg every 12 h) or placebo daily for 56 days. Primary outcomes were change in ppFEV₁ at Day 56 and safety. Other disease markers were evaluated.

Measurements and main results

Of 126 patients, 119 (94.4%) completed the study. Lumacaftor/ivacaftor was wel

SUBMITTER: Rowe SM 

PROVIDER: S-EPMC5461999 | biostudies-literature | 2017 Feb

REPOSITORIES: biostudies-literature