Lumacaftor/Ivacaftor in Patients Aged 6-11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR.

ABSTRACT:

Rationale

Combination lumacaftor/ivacaftor has been shown to improve lung function and other endpoints in patients aged 12 years and older with cystic fibrosis and homozygous for F508del-CFTR, but it has not been assessed in younger patients.

Objectives

In this open-label phase III trial, we evaluated the safety, tolerability, pharmacodynamics, and efficacy of lumacaftor/ivacaftor combination therapy in patients aged 6-11 years with cystic fibrosis who were homozygous for F508del-CFTR.

Methods

Patients (N = 58) received 200 mg lumacaftor/250 mg ivacaftor orally every 12 hours for 24 weeks in addition to their existing cystic fibrosis medications.

Measurements and main results

Lumacaftor/ivacaftor was well tolerated; the safety profile was generally similar to t

SUBMITTER: Milla CE 

PROVIDER: S-EPMC5440888 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature