Table I.
Clinical trials investigating immune checkpoint inhibitors monotherapy in hepatocellular carcinoma.
| First author, year | Therapy method | Target | Trial ID | Phase | Patient number | Lines of Therapy | Endpoint | Status | (Refs.) |
|---|---|---|---|---|---|---|---|---|---|
| El-Khoueiry et al, 2017 | Nivolumab (cohort 1) | PD-1 | NCT01658878 | I/II | 48 | First/second-line | DLT/MTD | Completed | (46) |
| El-Khoueiry et al, 2017 | Nivolumab (cohort 2) | PD-1 | NCT01658878 | I/II | 214 | First/second-line | ORR | Completed | (46) |
| El-Khoueiry et al, 2017 | Nivolumab (cohort 3) | PD-1 | NCT01658878 | I/II | 200 | First-line | ORR | Completed | (46) |
| Sangro et al, 2016 | Nivolumab vs. sorafenib | PD-1 | NCT02576509 | III | 726 | First-line | OS | Completed | (48) |
| CheckMate 459 | |||||||||
| Exposito et al, 2019 | Nivolumab vs. placebo | PD-1 | NCT03383458 | III | 530 | Adjuvant | PFS | Recruiting | (49) |
| CheckMate 9DX | |||||||||
| Zhu et al, 2018 | Pembrolizumab | PD-1 | NCT02702414 | II | 104 | Second-line | ORR/DCR | Completed | (50) |
| KEYNOTE-224 | |||||||||
| Finn et al, 2019 | Pembrolizumab vs. placebo | PD-1 | NCT02702401 | III | 413 | Second-line | OS/PFS | Recruiting | (25) |
| KEYNOTE-240 | |||||||||
| Hilmi et al, 2019 | Pembrolizumab | PD-1 | NCT03062358 | III | 450 | Second-line | OS | Recruiting | (72) |
| KETNOTE-394 | |||||||||
| Gao et al, 2019 | Pembrolizumab | PD-1 | NCT03211416 | I/II | 27 | First-line | ORR | Recruiting | (90) |
| Qin et al, 2020 | Camrelizumab | PD-1 | NCT02989922 | II | 220 | Second-line | ORR | Completed | (53) |
| Sangro et al, 2013 | Tremelimumab | CTLA-4 | NCT01008358 | II | 21 | First-line | DCR | Recruiting | (65) |
OS, overall survival; PFS, progression-free survival; DCR, disease control rate; ORR, objective response rate; DLT, dose limited toxicity; MTD, maximum tolerance dose.