Table 1.

Results reported from phase II/III clinical trials investigating immune checkpoint inhibitors in advanced BTC

Trial Name/Phase	Treatment Arms	Population	ORR	Median PFS	Median OS
NCT02829918 [24]   Phase 2	Nivolumab	Advanced BTC Second or third line N = 54	22%	3.7 months	14.2 months
KEYNOTE-158 [23,25] (NCT02628067)   Phase II	Pembrolizumab	Advanced MSS BTC Second line and beyond N = 104   Advanced MSI-H cholangiocarcinoma N = 22	MSS: 5.8%       MSI-H: 40.9%	2 months       4.2 months	9.1 months       24.3 months
CA209-538 [26] (NCT02923934)   Phase II	Nivolumab + ipilimumab	Advanced BTC First-line and beyond N = 39	23%	2.9 months	5.7 months
BilT-01 [27] (NCT03101566)   Phase II	Arm A: Nivolumab + Gem-Cis Arm B: Nivolumab + ipilimumab	Advanced BTC First-line N = 71	NR	Arm A: 7.4 months Arm B: 4.1 months	Arm A: 10.6 months Arm B: 8.3 months
NCT03046862 [28]   Phase II	Arm A: Gem-Cis → Gem-Cis + durvalumab Arm B: Gem-Cis + durvalumab Arm C: Gem-Cis + durvalumab + tremelimumab	Advanced BTC First-line N = 121	Arm A: 50.0% Arm B: 73.4% Arm C: 73.3%	Arm A: 13.0 months Arm B: 11.0 months Arm C: 11.9 months	Arm A: 15.0 months Arm B: 18.1 months Arm C: 20.7 months
NCT03895970 [29]   Phase II	Pembrolizumab, Lenvatinib	Advanced BTC Second line and beyond N = 32	25%	4.9 months	11.0 months
TOPAZ-1 [30,31]   Phase III	Durvalumab + Gemc-Cis vs. Placebo + Gem-Cis	Advanced, unresectable BTC First-line N = 341	ORR(durva vs. placebo) 26.7% vs. 18.7% OR(durva vs. placebo) 1.60 (95% CI 1.11−2.31)	mPFS(durva vs. placebo): 7.2 vs. 5.7 months HR(durva vs. placebo) 0.75 (95% CI 0.63−0.89) p = 0.001	mOS(durva vs. placebo) 12.9 vs. 11.3 months HR(durva vs. placebo) 0.76 (95% CI 0.64−0.91)

BTC, biliary tract cancer; CI, confidence interval; durva, durvalumab; Gem-Cis, gemcitabine-cisplatin; HR, hazard ratio; m, median; MSI-H, microsatellite instability high; MSS, microsatellite stable; NR, not reported; OR, odds ratio; ORR, overall survival; OS, overall survival; PFS, progression-free survival.