Results for CFQ-R Respiratory Domain Score1,2
Absolute change from baseline at Week 24 in CFQ-R Respiratory Domain score was a secondary endpoint in Trials 1 and 2 and was not statistically significant in either trial.3
- In a pooled analysis of Trials 1 and 2, the mean treatment difference for the CFQ-R Respiratory Domain score at Week 24 was 2.2 points vs placebo [95% CI (0.0, 4.5)]2*
- In the individual analyses of these trials, changes were not statistically significant with ORKAMBI vs placebo in either trial
- At Week 96 of the Extension Study, the LS mean absolute change from baseline in the CFQ-R Respiratory Domain score was 3.5 points [95% CI (1.3, 5.8)] for the ORKAMBI-to-ORKAMBI group and 0.5 points [95% CI (-2.7, 3.6)] for the placebo-to-ORKAMBI group1
*Results are based on a pooled analysis that was not prespecified and did not correct for multiple comparisons.
Trials 1 & 2 Study Design: Trials 1 and 2 were 24-week, Phase 3, randomized, double-blind, placebo-controlled trials of patients with CF who were homozygous for the F508del mutation and age 12 years and older. Patients received either ORKAMBI (lumacaftor 400 mg/ivacaftor 250 mg q12h) or placebo and continued to take their prescribed CF therapies.3
Extension Study Design: Patients who completed Trials 1 and 2 were eligible to enroll in a 96-week Extension Study. Of patients from Trials 1 and 2, 340 patients continued to receive ORKAMBI during the Extension Study and 176 rolled over from placebo to ORKAMBI.1