Baseline demographic and clinical characteristics of the study subjects
| Characteristic | Placebo n=17 | VX-809 | Total n=89 | |||
| 25 mg n=18 | 50 mg n=18 | 100 mg n=17 | 200 mg n=19 | |||
| Male, n (%) | 11 (65) | 9 (50) | 9 (50) | 12 (71) | 12 (63) | 53 (60) |
| Caucasian, n (%) | 17 (100) | 18 (100) | 18 (100) | 17 (100) | 19 (100) | 89 (100) |
| Age, years, median (range) | 28 (19–49) | 25.5 (18–50) | 24.5 (19–49) | 26 (18–54) | 25 (18–42) | 26 (18–54) |
| BMI, kg/m2, median (range) | 23 (19–31) | 22 (16–34) | 22 (19–31) | 23 (16–31) | 21 (19–27) | 22 (16–34) |
| FEV1 % predicted, median (range) | 78.4 (48.8–124.9) | 78.4 (34.2–104.5) | 61.5 (35.2–120.7) | 61.5 (40.0–128.3) | 68.2 (37.9–99.1) | 71 (34.2–128.3) |
| Sweat chloride, mmol/l, median (range) | 106.5 (80.0–125.5) | 100 (86.0–109.0) | 102.3 (76.0–120.0) | 106 (66.0–129.0) | 98.3 (72.0–122.5) | 103.5 (66.0–129.0) |
| NPD—chloride-free iso, mV, median (range) | 1.55 (−10.6* to 8.6) | 1.6 (−3.7 to 9.5) | 2.48 (−19.6* to 11.1) | 0.98 (−7.4 to 6.1) | 1.33 (−7.4 to 6.3) | 1.48 (−19.6 to 11.1) |
↵* High confidence analysis removed two outliers but did not change efficacy analysis.
BMI, body mass index; FEV1, forced expiratory volume in 1 s; NPD, nasal potential difference.