Table 1.
Patient baseline demographics and clinical characteristics.
| Parameter | Safety Run-in Phase | Expansion Phase | Overall (n = 25) | ||
|---|---|---|---|---|---|
| 40 mg/m2 Days 1–3 (n = 3) | 40 mg/m2 Day 1 + 66.8 mg/m2 Days 2, 3 (n = 6) | Total Run-in (n = 9) | 40 mg Days 1–3 (n = 16) | ||
| Median age, y (range) | 64.0 (63, 72) | 62.5 (53, 69) | 64.0 (53, 72) | 65.5 (51, 73) | 64.0 (51, 73) |
| n (%) ≤ 65 years | 2 (66.7%) | 4 (66.7%) | 6 (66.7%) | 8 (50.0%) | 14 (56.0%) |
| >65 years | 1 (33.3%) | 2 (33.3%) | 3 (33.3%) | 8 (50.0%) | 11 (44.0%) |
| Female:Male ratio | 0:3 | 2:4 | 2:7 | 5:11 | 7:18 |
| Race (n (%)) | |||||
| Asian | 0 | 0 | 0 | 1 (6.3%) | 1 (4.0%) |
| Black/African American | 0 | 0 | 0 | 2 (12.5%) | 2 (8.0%) |
| White | 1 (33.3%) | 5 (83.3%) | 6 (66.7%) | 10 (62.5%) | 16 (64.0%) |
| Other | 2 (66.7%) | 1 (16.7%) | 3 (33.3%) | 3 (18.8%) | 6 (24.0%) |
| ECOG performance status (n (%)) | |||||
| 0 | 1 (33.3%) | 2 (33.3%) | 3 (33.3%) | 5 (31.3%) | 8 (32.0%) |
| 1 | 2 (66.7%) | 4 (66.7%) | 6 (66.7%) | 11 (68.8%) | 17 (68.0%) |
| Child-Pugh-score (n (%)) | |||||
| A5 | 1 (33.3%) | 4 (66.7%) | 5 (55.6%) | 5 (31.3%) | 10 (40.0%) |
| A6 | 1 (33.3%) | 2 (33.3%) | 3 (33.3%) | 5 (31.3%) | 8 (32.0%) |
| B7 | 1 (33.3%) | 0 | 1 (11.1%) | 5 (31.3%) | 6 (24.0%) |
| B8 | 0 | 0 | 0 | 1 (6.3%) | 1 (4.0%) |
| Best response to previous sorafenib therapy (n (%)) | |||||
| CR | 0 | 0 | 0 | 0 | 0 |
| PR | 0 | 0 | 0 | 0 | 0 |
| SD | 2 (66.7%) | 4 (66.7%) | 6 (66.7%) | 9 (56.3%) | 15 (60.0%) |
| PD | 1 (33.3%) | 2 (33.3%) | 3 (33.3%) | 5 (31.3%) | 8 (32.0%) |
| Unknown | 0 | 0 | 0 | 2 (12.5%) | 2 (8.0%) |
Abbreviation: CR, complete response; PD, progressive disease; PR, partial response; SD, stable disease.