A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation



Status:Completed
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:12 - 65
Updated:4/21/2016
Start Date:April 2013
End Date:April 2014

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A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

The primary objective of the study was to evaluate the efficacy of lumacaftor in combination
with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF)
who are homozygous for the F508del mutation on the CF transmembrane conductance regulator
(CFTR) gene.

This was a Phase 3, randomized, double-blind, placebo-controlled, parallel-group multicenter
study of orally administered lumacaftor in combination with ivacaftor in participants aged
12 years and older with CF who are homozygous for the F508del-CFTR mutation.

The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1
[first dose of study drug] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7
days after the Week 24 Visit).

Inclusion Criteria:

- Confirmed diagnosis of CF

- Homozygous for the F508del CFTR mutation

- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent
(%) and less than or equal to (=<) 90% of predicted normal for age, sex, and height

- Willing to remain on a stable CF medication regimen through Week 24 or, if
applicable, the Safety Follow up Visit

Exclusion Criteria:

- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 4 weeks before first
dose of study drug

- History of solid organ or hematological transplantation

- History of alcohol or drug abuse in the past year

- Ongoing or prior participation in an investigational drug study (including studies
investigating lumacaftor and/or ivacaftor) within 30 days of screening.

- Use of strong inhibitors, moderate inducers, or strong inducers of Cytochrome P450 3A
(CYP3A) within 14 days before Day 1 of dosing
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