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Study designs


CheckMate 9DW (Phase 3)1

  • Phase 3, randomised, open-label study investigating nivolumab plus ipilimumab in a first line setting in patients with unresectable HCC without previous systemic therapy1
  • Primary endpoints: OS1
  • Secondary endpoints: ORR, DOR, TTD1
  • Exploratory endpoints: safety, PFS, TTR per BICR, ORR and PFS on next-line therapy, HRQoL, influence of PD-L1 on overall survival1

Figure adapted from Yau T, et al 2025.1

CheckMate 040 Cohort 4 (Phase 1/2)2

  • Phase 1/2, dose-escalation, open-label study in patients with advanced HCC2
  • Primary endpoints: Safety and tolerability using NCI CTCAE v4.0, ORR and DOR by investigator assessmenta,2
  • Secondary endpoints: DCR, PFS, OS, TTP, TTR2
  • Other key endpoints: BOR and ORR based on BICR-assessed tumor responsea

The FDA indication is only for patients previously treated with sorafenib.
**Only Arm A has been approved by the FDA.

aUsing RECIST v1.1.

Figure adapted from Yau et al. 2020.2

BICR, blinded independent central review; BID, twice a day; BOR, best overall response; DCR, disease control rate; DOR, duration of response; ECOG PS, Eastern Cooperative Oncology Group Performance Status; HBV, hepatitis B virus; HCC, hepatocellular carcinoma; HCV, hepatitis C virus; HRQoL, health-related quality of life; IPI, ipilimumab; LRT, locoregional therapy; NCI CTCAE, National Cancer Institute Common Terminology Criteria for Adverse Events; NIVO, nivolumab; ORR, overall response rate; OS, overall survival; PD-L1, programmed death-ligand 1; PFS, progression-free survival; PO, oral administration; QD, once a day; QxW, every x weeks; TTP, time to progression; TTD, time to deterioration; TTR, time to response.

1. Yau T, et al. The Lancet 2025;405:1851-1864.

2. Yau T, et al. JAMA Oncol 2020;6:e204564.