TABLE 1.
Study Design and Baseline Characteristics for GOAL and ENVISION Studies
| Characteristic | GOAL | ENVISION | |
|---|---|---|---|
| Ivacaftor (n = 35) | Placebo (n = 25) | Ivacaftor (n = 23) | |
| Trial design | Observational | Randomized | |
| Control | Predrug data | Placebo arm | |
| Study visits analyzed | Baseline and 3 and 6 months postbaseline | Baseline and 24 and 48 weeks postbaseline | |
| Sex (male/female), n | 19/16 | 16/9 | 9/14 |
| Age, y | |||
| Mean (SD) | 8.7 (1.6) | 8.8 (1.8) | 8.5 (1.8) |
| Range | 6–11 | 6–11 | 6–11 |
| Height, mean (SD), cm | 131.1 (11.3) | 131.9 (12.0) | 133.2 (14.3) |
| Height z score, mean (SD) | −0.15 (1.3) | −0.34 (0.9) | 0.00 (1.0) |
| Weight, mean (SD), kg | 29.8 (7.2) | 29.6 (7.0) | 31.2 (10.3) |
| Weight z score, mean (SD) | 0.01 (1.2) | −0.16 (0.8) | 0.08 (1.0) |
| BMI, mean (SD), kg/m2 | 17.1 (2.4) | 16.8 (1.8) | 17.2 (2.7) |
| FEV1, mean (SD), % predicted | 106.4 (14.6) | 83.8 (20.8) | 87.3 (14.6) |
| Pseudomonas positive,a n (%) | 15 (42.9) | 12b (57.1) | 8c (40.0) |
| Sweat chloride, mean (SD), mmol/L | 107.3 (9.9) | 104.3 (8.7) | 104.2 (15.2) |
a
Within 12 months of starting the study.
b
Of 21 patients with available data.
c
Of 20 patients with available data.