Table 5 Efficacy assessed by IRC and the investigator in the dose-expansion stage.
Assessment | All patients (n = 22) | CTFI < 90 days (n = 8) | CTFI ≥ 90 days (n = 14) | |||
|---|---|---|---|---|---|---|
IRC | Investigator | IRC | Investigator | IRC | Investigator | |
RECIST responses | ||||||
 Complete response | 0 | 0 | 0 | 0 | 0 | 0 |
 Partial response | 10 (45.5%) | 10 (45.5%) | 3 (37.5%) | 3 (37.5%) | 7 (50.0%) | 7 (50.0%) |
 Stable disease | 10 (45.5%) | 8 (36.4%) | 5 (62.5%) | 3 (37.5%) | 5 (35.7%) | 5 (35.7%) |
 Progressive disease | 1 (4.5%) | 3 (13.6%) | 0 | 2 (25.0%) | 1 (7.1%) | 1 (7.1%) |
 Not evaluable | 1 (4.5%) | 1 (4.5%) | 0 | 0 | 1 (7.1%) | 1 (7.1%) |
 Overall response, % (95% CI) | 45.5% (26.9–65.3) | 45.5% (26.9–65.3) | 37.5% (13.7–69.4) | 37.5% (13.7–69.4) | 50.0% (26.8–73.2) | 50.0% (26.8–73.2) |
 Disease control, % (95% CI) | 90.9% (72.2–97.5) | 81.8% (61.5–92.7) | 100.0% (67.6–100.0) | 75.0% (40.9–92.9) | 85.7% (60.1–96.0) | 85.7% (60.1–96.0) |
Duration of response | ||||||
 Disease progression, relapse, or death events in responding patients, n/N (%) | 4/10 (40.0%) | 7/10 (70.0%) | 1/3 (33.3%) | 1/3 (33.3%) | 3/7 (42.9%) | 6/7 (85.7%) |
 Censored*, n/N (%) | 6/10 (60.0%) | 3/10 (30.0%) | 2/3 (66.7%) | 2/3 (66.7%) | 4/7 (57.1%) | 1/7 (14.3%) |
 Median duration of response, months (95% CI) | 4.2 (2.7–inf) | 2.9 (2.8–inf) | NR (4.0, –) | NR (2.9–inf) | 4.2 (2.7–inf) | 2.9 (2.8–inf) |
Progression-free survival | ||||||
 Progression-free survival events, n (%) | 11 (50.0%) | 17 (77.3%) | 4 (50.0%) | 5 (62.5%) | 7 (50.0%) | 12 (85.7%) |
 Censored*, n (%) | 11 (50.0%) | 5 (22.7%) | 4 (50.0%) | 3 (37.5%) | 7 (50.0%) | 2 (14.3%) |
 Median progression-free survival, months (95% CI) | 5.6 (4.1–6.9) | 4.2 (4.0–5.4) | 6.6 (3.0–inf) | 4.4 (0.5–inf) | 5.4 (4.1–inf) | 4.2 (4.0–5.4) |
Overall survival | ||||||
 Deaths | 5 (22.7%) | 3 (37.5%) | 2 (14.3%) | |||
 Censored* | 17 (77.3%) | 5 (62.5%) | 12 (85.7%) | |||
 Median overall survival, months (95% CI) | 11.0 (9.2–inf) | 9.2 (3.0–inf) | 11.0 (inf–inf) | |||
