Table 1. Baseline patient demographics and characteristics by the MTD of lomitapide at Day 1 and in the overall FAS populationa.
| Characteristic | Lomitapide MTD at Visit 1 |
FAS (N = 5) |
||
|---|---|---|---|---|
| 10 mg (n = 1) | 20 mg (n = 3) | 40 mg (n = 1) | ||
| Age (years) | ||||
| Mean (SD) | 47.0 | 50.7 (14.22) | 53.0 | 50.4 (10.29) |
| Median | 47.0 | 44.0 | 53.0 | 47.0 |
| (Min., Max.) | (41, 67) | (41, 67) | ||
| Gender, n (%) | ||||
| Male | 1 (100.0) | 2 (66.7) | 0 | 3 (60.0) |
| Female | 0 | 1 (33.3) | 1 (100.0) | 2 (40.0) |
| Ethnicity, n (%) | ||||
| Japanese | 1 (100.0) | 3 (100.0) | 1 (100.0) | 5 (100.0) |
| Weight (Day 1) (kg) | ||||
| Mean (SD) | 78.50 | 55.10 (10.213) | 41.20 | 57.00 (15.259) |
| Median | 78.50 | 57.20 | 41.20 | 57.20 |
| (Min., Max.) | (44.0, 64.1) | (41.2, 78.5) | ||
| BMI (Day 1) (kg/m2) | ||||
| Mean (SD) | 29.10 | 20.10 (2.615) | 17.10 | 21.30 (4.911) |
| Median | 29.10 | 18.90 | 17.10 | 18.90 |
| (Min., Max.) | (29.1, 29.1) | (18.3, 23.1) | (17.1, 17.1) | (17.1, 29.1) |
| Use of Apheresis at Day 1, n | 1 | 3 | 1 | 5 |
| Yes | 0 | 3 (100.0) | 1 (100.0) | 4 (80.0) |
| No | 1 (100.0) | 0 | 0 | 1 (20.0) |
| Frequency of Apheresis at Day 1, n | 0 | 3 | 1 | 4 |
| Every week | 0 | 2 (66.7) | 0 | 2 (50.0) |
| Every 2 weeks | 0 | 1 (33.3) | 1 (100.0) | 2 (50.0) |
BMI, body mass index; FAS, full analysis set; MTD, maximum tolerated dose; SD, standard deviation
a
Full analysis set and safety population