Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ∆F508-CFTR Gene Mutation

The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

This was a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.

Inclusion Criteria:

• Confirmed diagnosis of CF with ∆F508-CFTR mutation in both alleles
• Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) of predicted normal for age, gender, and height
• Weight >=40 kilograms (kg) and body mass index greater than or equal to 18.5 kilogram per square meter (kg/m^2)
• Screening laboratory values, tests, and physical examination within acceptable ranges
• Negative pregnancy test (for women of child-bearing potential)
• Able and willing to follow contraceptive requirements
• Willing to remain on a stable medication regimen for the duration of study participation

Exclusion Criteria:

• History of any illness, or any ongoing acute illness, that could impact the safety of the study participant or may confound results of study
• Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug
• Impaired hepatic or renal function
• History of organ or hematological transplant