Table 1.
Past clinical trials on systemic treatments in HCC that resulted in regulatory approval.
| Trial Name | Treatment Arms | Line of Therapy | Primary Endpoint | ORR | PFS | OS |
|---|---|---|---|---|---|---|
| SHARP [5] | Sorafenib vs. Placebo | First | OS | 2 vs. 1% | 5.5 vs. 2.8 months | 10.7 vs. 7.9 months (HR 0.69) |
| REFLECT [20] | Lenvatinib vs. Sorafenib | First | OS | 24.1 vs. 9.2% | 7.4 vs. 3.7 months | 13.6 vs. 12.3 months (HR 0.92) |
| IMbrave150 [25,26] | Atezolizumab+bevacizumab vs. sorafenib | First | OS and PFS | 29.8 vs. 11.3% | 6.8 vs. 4.3 months | 19.2 vs. 13.4 months (HR 0.66) |
| RESORCE [21] | Regorafenib vs. Placebo | Second | OS | 11 vs. 4% | 3.1 vs. 1.5 months | 10.6 vs. 7.8 months (HR 0.63) |
| CELESTIAL [22] | Cabozantinib vs. Placebo | Second and third | OS | 4 vs. 1% | 5.2 vs. 1.9 months | 10.2 vs. 8.0 months (HR 0.76) |
| REACH-2 [24] | Ramucirumab vs. Placebo (in patients with AFP > 400 ng/mL) | Second | OS | 5 vs. 1% | 2.8 vs. 1.6 months | 8.5 vs. 7.3 months (HR 0.71) |
| CHECKMATE 040 [16] | Nivolumab * single arm | Second | ORR | 15% | N/A | N/A |
| KEYNOTE 224 [17] | Pembrolizumab * single arm | Second | ORR | 17% | N/A | N/A |
| CHECKMATE 040 [27] | Nivolumab + ipilimumab * single arm | Second | ORR | 32% | N/A | N/A |
* Regulatory approval by the U.S. Food and Drug Administration but not the European Medicines Agency. HR, hazard ratio; N/A, not available; ORR, overall response rate; OS, overall survival; PFS, progression-free survival.