TRIAL 2: A double-blind, active-controlled, head-to-head Phase 3 trial in patients homozygous for F508del mutation1,2
Prior to the run-in period, all patients in this study discontinued previous CFTR modulator therapies but remained on their standard-of-care CF therapies.1
aAll patients who completed the study were eligible to roll over into a 192-week, open-label Extension Study.1,3
Key inclusion criteria1,2b
- Confirmed CF diagnosis, clinically stable, and at least 12 years of age
- Homozygous for the F508del mutation
- ppFEV1 between 40% and 90% at screening
bKey exclusion criteria included clinically significant cirrhosis with or without portal hypertension, a history of colonization with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus), and solid organ or hematologic transplantation.1,4
cA hierarchical testing procedure was performed for key secondary endpoints. For an endpoint to be significant, both it and all previous tests in the hierarchy had to achieve P<0.05.2
Summary of Efficacy Results
Significant improvements in lung function and sweat chloride vs active comparator1,2
Significantly improved CFQ-R Respiratory Domain score vs active comparator1,2
BMI, body mass index; CF, cystic fibrosis; CFTR, cystic fibrosis transmembrane conductance regulator; CFQ-R, Cystic Fibrosis Questionnaire-Revised; CI, confidence interval; IV, intravenous; LS, least square; MCID, minimal clinically important difference; ppFEV₁, percent predicted forced expiratory volume in 1 second; q12h, every 12 hours; qam, every morning; qpm, every evening; RR, rate ratio, SE, standard error; SwCl, sweat chloride; WK, week.