Table 2. Trial Features Associated With Positive vs Negative Results in Randomized Trials That Had Primary Completion Dates Before December 2022.
| Characteristic | Trials, No. (%) | P valuea | |
|---|---|---|---|
| Positive (n = 2) | Negative (n = 17) | ||
| Sample size, No. | |||
| <50 | 0 | 2 (11.8) | .57 |
| 50-200 | 0 | 8 (47.1) | |
| >200 | 2 (100) | 7 (42.2) | |
| Phases | |||
| I-II or II | 0 | 13 (76.5) | .08 |
| II-III or III | 2 (100) | 4 (23.5) | |
| Setting | |||
| Neoadjuvant | 1 (50) | 3 (17.7) | .46 |
| Adjuvant | 0 | 1 (5.9) | |
| Metastatic | 1 (50) | 13 (76.5) | |
| Primary end pointb | |||
| pCR | 1 (25) | 3 (12) | .67 |
| ORR | 0 | 4 (16) | |
| PFS or EFS or IDFS | 1 (25) | 10 (40) | |
| OS | 1 (25) | 2 (8) | |
| Othersc | 1 (25) | 6 (24) | |
| Lead sponsor | |||
| Industry | 2 (100) | 11 (64.7) | >.99 |
| NIH | 0 | 0 | |
| Other | 0 | 6 (35.3) | |
| Center | |||
| Single-center | 0 | 2 (11.8) | >.99 |
| Multicenter | 2 (100) | 14 (82.3) | |
| NA | 0 | 1 (5.9) | |
Abbreviations: EFS, event-free survival; IDFS, invasive disease-free survival; NIH, National Institutes of Health; ORR, overall response rate; OS, overall survival; pCR, pathological complete response; PFS, progression-free survival.
a
P values were calculated with Fisher test.
b
Co-primary end points are counted separately.
c
Others include safety, tumor-infiltrating lymphocytes increase, clinical benefit rate, and circulating tumor DNA clearance.