A phase 3, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis who have the R117H-CFTR mutation.
This phase 3 trial (NCT01614457) was conducted in 35 sites in 2 countries (UK and USA) to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis who have the R117H-CFTR mutation. Sixty-nine CF patients (≥ 6 years-old) with the R117-CFTR mutation on at least one allele were enrolled to evaluate the safety and efficacy of ivacaftor (150 mg q12h or placebo) in a 24-week, double-blind, placebo-controlled parallel-group study. The results showed that 24 weeks of ivacaftor treatment did not result in significant change in percent predicted FEV1 and BMI, as compared with placebo. It did result in significant improvement of CFQ-R (+8.4, p=0.009) and sweat chloride (-24 mmol/L, p<0.001). In the subgroup of patients ≥ 18 years-old (n=50), ivacaftor treatment induced a significant difference in FEV1 (+5.0 percentage points of absolute change in % predicted, as compared with placebo, p=0.01). Ivacaftor was generally well tolerated. In conclusion, although the study did not meet its primary endpoint, secondary endpoints and subgroup analyses suggest that ivacaftor may benefit some patients with R117-CFTR, particularly those aged ≥18.
1 ECFS-CTN site in UK participated in this study and enrolled 13 of the 69 patients.
The summary of data provided here are from the presentation of the study results at the European Cystic Fibrosis Conference in Gothenburg (Sweden) in June 2014.