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Table 1 Clinical trials of systemic therapy in HCC

From: Emerging treatment modalities for systemic therapy in hepatocellular carcinoma

Therapy Time Trial Line Drug Control Primary outcomes (HR; 95% CI)
Targeted therapy
  2005 NCT00033462 First Erlotinib None 6-month PFS rate: 32%
  2007 SHARP First Sorafenib Placebo OS: 10.7 vs. 7.9 (0.69; 0.55–0.87)
  2008 Asia-Pacific First Sorafenib Placebo OS: 6.5 vs. 4.2 (0.68; 0.50–0.93)
  2011 NCT00699374 First Sunitinib Sorafenib OS: 7.9 vs. 10.2 (1.3; 1.13–1.5)
  2011 NCT00604721 First Selumetinib None TTP: 2
  2012 BRISK-FL First Brivanib Sorafenib OS: 9.5 vs. 9.9 (1.06; 0.93–1.22)
  2012 BRISK-PS Second Brivanib Placebo OS: 9.4 vs. 8.2 (0.89; 0.69–1.15)
  2013 NCT00427973 First Cediranib None 3-month PFS rate: 77%
  2014 EVOLVE-1 Second Everolimus Placebo OS: 7.6 vs. 7.3 (1.05; 0.86–1.27)
  2015 NCT01210495 Second Axitinib None 16-week DCR rate: 42.3%
  2015 REACH Second Ramucirumab Placebo OS: 9.2 vs. 7.6 (0.87; 0.72–1.05)
  2016 NCT01232296 First Dovitinib Sorafenib OS: 8.0 vs. 8.4 (1.27; 0.90–1.79)
  2017 RESORCE Second Regorafenib Placebo OS: 10.6 vs. 7.8 (0.63; 0.50–0.79)
  2018 REFLECT First Lenvatinib Sorafenib OS: 13.6 vs. 12.3 (0.95; 0.79–1.06)
  2018 NCT01004003 First Nintedanib Sorafenib TTP: 5.5 vs. 4.6 (1.44; 0.81–2.57)
  2018 NCT01915589 First Refametinib None ORR: 0%; DCR: 56.3%
  2018 METIV-HCC Second Tivantinib Placebo OS: 8.4 vs. 9.1 (0.97; 0.75–1.25)
  2019 CELESTIAL Second Cabozantinib Placebo OS: 10.2 vs. 8.0 (0.76; 0.63–0.92)
  2019 REACH-2 Second Ramucirumab Placebo OS: 8.5 vs. 7.3 (0.71; 0.531–0.949)
  2020 NCT02645981 First Donafenib Sorafenib OS: 12.1 vs. 10.3 (0.831; 0.699–0.988)
  2020 NCT02329860 Second Apatinib Placebo OS: 8.7 vs. 6.8 (0.785; NA)
Immunotherapy
  2013 NCT01008358 First Tremelimumab None PR: 17.6%; DCR: 76.4%
  2017 Checkmate-040 Second Nivolumab None ORR: ~ 17%
  2018 KEYNOTE-224 Second Pembrolizumab None ORR: 17%
  2019 Checkmate-459 First Nivolumab Sorafenib OS: 16.4 vs. 14.7 (0.85; 0.72–1.02)
  2019 KEYNOTE-240 Second Pembrolizumab Placebo PFS: 3.0 vs. 2.8 (0.718; 0.570–0.904)
  2020 NCT02989922 Second Camrelizumab None ORR: 14.7%
  1. OS, overall survival; PFS, progression-free survival; DCR: disease control rate; TTP: time to progression; PR: partial response; ORR: object response rate; HR, hazard ratio; CI, confidence interval
  2. * Primary outcomes (months or rate)