Table 3.
Secondary Efficacy Endpoints from Part B
| ELX/TEZ/IVA |
|||
|---|---|---|---|
| All Patients (N = 66)* | F/MF (N = 37) | F/F (N = 29) | |
| ppFEV1, percentage points | |||
| Baseline, mean (SD)† | 88.8 (17.7) | 89.8 (17.5) | 87.3 (18.3) |
| Absolute change through Week 24, LS mean (95% CI) | 10.2 (7.9 to 12.6)‡ | 9.1 (6.3 to 11.9)‡ | 11.2 (7.2 to 15.2)‡ |
| CFQ-R respiratory domain score, points | |||
| Baseline, mean (SD)† | 80.3 (15.2) | 79.1 (17.3) | 81.8 (12.0) |
| Absolute change through Week 24, LS mean (95% CI) | 7.0 (4.7 to 9.2)‡ | 6.9 (3.2 to 10.6)‡ | 7.0 (3.9 to 10.1)‡ |
| LCI2.5, units | |||
| Baseline, mean (SD)† | 9.77 (2.68) | 9.34 (1.82) | 10.26 (3.36) |
| Absolute change through Week 24, LS mean (95% CI) | −1.71 (−2.11 to −1.30)‡ | −1.72 (−2.11 to −1.33)‡ | −1.64 (−2.34 to −0.94)‡ |
| Sweat chloride, mmol/L | |||
| Baseline, mean (SD)† | 102.2 (9.1) | 104.4 (7.2) | 99.3 (10.8) |
| Absolute change through Week 24, LS mean (95% CI) | −60.9 (−63.7 to −58.2)‡ | −55.1 (−59.0 to −51.2)‡ | −70.4 (−75.6 to −65.3)‡ |
Definition of abbreviations: CFQ-R = Cystic Fibrosis Questionnaire–Revised; CFTR = cystic fibrosis transmembrane conductance regulator; CI = confidence interval; ELX/TEZ/IVA = elexacaftor/tezacaftor/ivacaftor; F/F = homozygous for the F508del-CFTR mutation; F/MF = heterozygous for the F508del-CFTR mutation and a minimal function CFTR mutation; LCI2.5 = lung clearance index2.5; LS = least squares; ppFEV1 = percentage of predicted FEV1.
N indicates the total number of patients in each population; LS means were based on mixed-effects model for repeated measures; absolute changes through Week 24 were averages of the visits between Weeks 4 and 24 (inclusive).
All patients in the full analysis set.
Baseline was defined as the most recent nonmissing measurement before the first dose of study drug.
Nominal P value <0.001.