Table 1. Patient Demographics and Baseline Characteristics (ITT Population).
| Characteristic | Treatment Arm | |
|---|---|---|
|
| ||
| Ataluren | Placebo | |
|
| ||
| N=116 | N=116 | |
|
| ||
| Age, years | ||
| Mean (SD) | 22.8 (10.18) | 23.2 (9.32) |
| Median | 22.0 | 22.0 |
| Range | 6, 49 | 8, 53 |
|
| ||
| Sex, n (%) | ||
| Male | 60 (51.7%) | 58 (50.0%) |
| Female | 56 (48.3%) | 58 (50.0%) |
|
| ||
| Body weight, kg | ||
| Mean (SD) | 53.5 (13.94) | 56.0 (13.15) |
| Median | 54.4 | 57.2 |
| Range | 21, 105 | 24, 93 |
|
| ||
| % predicted FEV1 at baseline | ||
| Mean (SD) | 62.1 (13.62) | 60.2 (15.14) |
| Median | 63.4 | 59.0 |
| Range | 38.4, 90.3 | 36.2, 92.6 |
|
| ||
| Sweat chloride at baselinea | N=114 | N=111 |
| Mean (SD) | 100.1 (14.22) | 96.6 (15.93) |
| Median | 101.5 | 100.0 |
| Range | 22.5, 128.0 | 22.0, 117.5 |
|
| ||
| Inhaled antibiotic use at randomizationb | 64 (55.2%) | 63 (53.4%) |
|
| ||
| Aminoglycoside (tobramycin) | 44 (37.9%) | 42 (35.6%) |
|
| ||
| Colistin | 30 (25.9%) | 22 (18.6%) |
|
| ||
| Aztreonam | 10 (8.3%) | 8 (6.8%) |
a
The results of historical sweat chloride tests were used for eligibility assessment of 2 patients whose centrally analyzed baseline sweat chloride value was subsequently found to be <40 mEq/L.
b
A patient was considered to be using inhaled antibiotics at baseline even if the patient was in the “off” portion of an intermittent cycling regimen. Patients may have been using >1 antibiotic at baseline.
Note: There were no statistical differences between the 2 arms
Abbreviations: FEV1 = forced expiratory volume in 1 second, SD = standard deviation