Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

A Phase 3, Open-label Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study to evaluate the pharmacokinetics, safety, and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the F508del-mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Inclusion Criteria:

• Confirmed diagnosis of CF defined as: with 2 CF-causing mutations, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
• Subjects who weigh ≥15 kg without shoes at Screening Visit
• Subjects who are homozygous for the F508del-CFTR mutation
• Subjects with percent predicted forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) (Part A) or ≥40% (Part B) at Screening Visit where the predicted values are adjusted for age, sex, and height using the Wang equation
• Subjects with stable CF disease and who are willing to remain on stable CF medication regimen
• Able to swallow tablets

Exclusion Criteria:

• History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
• Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 of the study
• Abnormal liver function as defined in the protocol at Screening Visit
• Abnormal renal function as defined in the protocol at Screening Visit
• History of solid organ or hematological transplantation
• Ongoing participation in an investigational drug study or prior participation in an investigational drug study within 30 days prior of Screening Visit
• History or evidence of lens opacity or cataract at Screening Visit
• Colonization with organisms associated with a more rapid decline in pulmonary status at Screening Visit (Part A only)
• A standard 12-lead ECG demonstrating QTcF >450 msec at Screening Visit