Table 1. Baseline characteristics of study participants.
| Characteristic | Part 1 | Part 2 | ||||||||
| Placebo (n = 4) | Ivacaftor | TOTAL (n = 20) | Placebo (n = 4) | Ivacaftor | TOTAL (n = 19) | |||||
| 25 mg/75 mg (n = 4) | 75 mg/25 mg (n = 4) | 75 mg/150 mg (n = 4) | 150 mg/75 mg (n = 4) | 150 mg (n = 8) | 250 mg (n = 7) | |||||
| Sex, n (%) | ||||||||||
| Male | 2 (50) | 1 (25) | 4 (100) | 1 (25) | 1 (25) | 9 (45) | 3 (75) | 3 (38) | 4 (57) | 10 (53) |
| Female | 2 (50) | 3 (75) | 0 | 3 (75) | 3 (75) | 11 (55) | 1 (25) | 5 (63) | 3 (43) | 9 (47) |
| Race, n (%) | ||||||||||
| White | 4 (100) | 4 (100) | 4 (100) | 4 (100) | 4 (100) | 20 (100) | 4 (100) | 8 (100) | 7 (100) | 19 (100) |
| Age, yr, median (range) | 36 (19–48) | 31 (22–51) | 41 (22–50) | 26 (19–34) | 21 (19–33) | 30 (19–51) | 24 (18–42) | 23 (18–40) | 21 (20–38) | 21 (18–42) |
| BMI, kg/m2, median (range) | 23 (22–29) | 23 (20–24) | 24 (19–27) | 20 (19–24) | 21 (17–26) | 23 (17–29) | 22 (21–23) | 22 (20–23) | 23 (20–25) | 22 (20–25) |
| CFTR genotype | ||||||||||
| G551D/F508del | 3 (75) | 4 (100) | 4 (100) | 2 (50) | 3 (75) | 16 (80) | 4 (100) | 7 (88) | 5 (71) | 16 (84) |
| G551D/1078delT | 1 (25) | – | – | – | – | 1 (5) | – | – | – | – |
| G551D/G551D | – | – | – | – | 1 (25) | 1 (5) | – | – | – | – |
| G551D/N1303K | – | – | – | 1 (25) | – | 1 (5) | – | – | – | – |
| G551D/R553X | – | – | – | 1 (25) | – | 1 (5) | – | – | – | – |
| G551D/3849+10 kbC | – | – | – | – | – | – | – | – | 1 (14) | 1 (5) |
| G551D/6214→1G+7T | – | – | – | – | – | – | – | 1 (13) | – | 1 (5) |
| G551D/G542X | – | – | – | – | – | – | – | – | 1 (14) | 1 (5) |
| % Predicted FEV1, median (range) | ||||||||||
| 40% to <70%, n (%) | 3 (75) | 3 (75) | 4 (100) | 2 (50) | 4 (100) | 16 (80) | 2 (50) | 5 (63) | 3 (43) | 10 (53) |
| 70% to <90%, n (%) | – | – | – | 1 (25) | – | 1 (5) | ||||
| >90%, n (%) | – | 1 (25) | – | 1 (25) | – | 3 (15) | 2 (50) | 1 (13) | 1 (14) | 4 (21) |
| Sweat chloride, mmol/L, median (range) | 105.25 (97.00–112.00) | 107.50 (60.00– 117.00) | 104.50 (102.00–117.00) | 97.50 (92.00–102.50) | 99.00 (83.00–104.00) | 102.00 (60.00–117.00) | 93.75 (88.00–109.50) | 100.13 (86.75–112.50) | 97.25 (84.75–115.75) | 95.50 (84.75–115.75) |
BMI, body-mass index; CFTR, cystic fibrosis transmembrane conductance regulator; FEV1, forced expiratory volume in one second.