Table 3.
Summary of efficacy endpoints
| Response, % | Intent-to-treat population, N = 48 | Declared dose cohort, N = 42 |
|---|---|---|
| Objective response rate (90% CI; 95% CI) | 10.4% (3.5–22.3%; 4.2–20.7%) | 11.9% (4.8–23.4%; 3.9–25.6%) |
| CR | 4.1% (0.5–14.2%; 0.7–12.5%) | 4.8% (0.9–14.2%; 0.6–16.1%) |
| PR | 6.3% (1.3–17.1%; 1.7–15.3%) | 7.1% (1.9–17.4%; 1.5–19.4%) |
| SD (95% CI) | 45.8% (31.3–60.8%) | 42.9% (27.7–59.0%) |
| PD (95% CI) | 43.8% (29.4–58.8%) | 45.2% (29.8–61.3%) |
| Clinical benefit rate (95% CI) | 56.2% (41.1–70.5%) | 54.8% (38.7–70.2%) |
| Survival | ||
| 12 month overall survival (95% CI) | 52.7% (40.1–69.2%) | 53.1% (36.8–67.0%) |
| Overall survival (95% CI) | 12.5 months (10.8–14.6 months) | 12.5 months (10.2–13.0 months) |
CR denotes complete response; PR denotes partial response; SD denotes stable disease; PD denotes progressive disease. The primary efficacy endpoint was objective response rate, and the two secondary efficacy endpoints were clinical benefit rate and 12 month overall survival.