Table 1.
Demographics and baseline characteristics (mITT population).
| Variable | ALIS 590 mg Once Daily N=148 |
TIS 300 mg Twice Daily N=146 |
Total N=294 |
|---|---|---|---|
| Race/ethnicity, n (%) | |||
| White (not of Hispanic origin) | 139 (93.9) | 141 (96.6) | 280 (95.2) |
| Hispanic | 5 (3.4) | 3 (2.1) | 8 (2.7) |
| African | 1 (0.7) | 0 | 1 (0.3) |
| Asian | 0 | 0 | 0 |
| Other | 3 (2.0) | 1 (0.7) | 4 (1.4) |
| Not reported | 0 | 1 (0.7) | 1 (0.3) |
| Sex, n (%) | |||
| Male | 79 (53.4) | 76 (52.1) | 155 (52.7) |
| Female | 69 (46.6) | 70 (47.9) | 139 (47.3) |
| Age | |||
| Mean (SD) | 22.8 (10.2) | 22.0 (10.0) | 22.4 (10.1) |
| 6–12 years, n (%) | 27 (18.2) | 26 (17.8) | 53 (18.0) |
| 13–18 years, n (%) | 34 (23.0) | 33 (22.6) | 67 (22.8) |
| >18 years, n (%) | 87 (58.8) | 87 (59.6) | 174 (59.2) |
| Height, mean (SD), cm | 162.3 (15.0) | 162.2 (15.6) | 162.3 (15.2) |
| Weight, mean (SD), kg | 54.5 (17.2) | 53.1 (15.9) | 53.8 (16.5) |
| BMI, mean (SD), kg/m2 | 20.1 (4.0) | 19.7 (3.7) | 19.9 (3.9) |
| CF genotype, n (%) | |||
| ΔF508 homozygous | 72 (48.6) | 70 (47.9) | 142 (48.3) |
| ΔF508 heterozygous | 40 (27.0) | 43 (29.5) | 83 (28.2) |
| Other | 21 (14.2) | 25 (17.1) | 46 (15.6) |
| FEV1% predicted | |||
| n | 148 | 144 | 292 |
| Mean (SD) | 64.5 (21.5) | 61.9 (22.0) | 63.2 (21.7) |
| FEV1% predicted stratification at screening for randomization | |||
| 25%–50%, n (%) | 42 (28.4) | 44 (30.1) | 86 (29.3) |
| >50%–75%, n (%) | 54 (36.5) | 55 (37.7) | 109 (37.1) |
| >75%, n (%) | 52 (35.1) | 47 (32.2) | 99 (33.7) |
| FEV1 (L) | |||
| n | 148 | 144a | 292 |
| Mean (SD) | 2.142 (0.861) | 2.037 (0.846) | 2.090 (0.854) |
| Use of bronchodilator | 113 (76.4) | 111 (76.0) | 224 (76.2) |
| Use of azithromycin | 76 (51.4) | 73 (50.0) | 149 (50.7) |
a
Baseline FEV1 data were not available for 2 patients.
ALIS, amikacin liposome inhalation suspension; BMI, body mass index; CF, cystic fibrosis; FEV1, forced expiratory volume in 1 second; mITT, modified intention-to-treat; TIS, tobramycin inhalation solution.