Table 2.
On-going clinical trials.
| Study Name | Agent | Target | Phase | Setting | Number | Primary Outcomes |
|---|---|---|---|---|---|---|
| ICIs plus chemotherapy | ||||||
| NCT03260712 | cisplatin/gemcitabine + pembrolizumab |
PD-1 | II | 1 line | 50 | PFS rate at 6 months |
| NCT04003636 | cisplatin/gemcitabine + pembrolizumab vs. cisplatin/gemcitabine |
PD-1 | III | 1 line | 788 | OS |
| NCT03875235 | cisplatin/gemcitabine + durvalumab vs. cisplatin/gemcitabine |
PD-L1 | III | 1 line | 757 | OS |
| NCT03478488 | KN035 plus gemcitabine/ oxaliplatin vs. gemcitabine/oxaliplatin |
PD-L1 | III | 1 line | 480 | OS |
| NCT04172402 | Nivolumab + S-1 + gemcitabine | PD-1 | II | 1 line | 48 | ORR |
| NCT04027764 | Toripalimab + S-1 + nab-paclitaxel | PD-1 | II | 1 line | 30 | ORR |
| NCT03796429 | Toripalimab + S-1 + gemcitabine | PD-1 | II | 1 line | 40 | PFS OS |
| NCT04191343 | Toripalimab + gemcitabine + oxaliplatin |
PD-1 | II | 1 line | 20 | ORR |
| NCT03785873 | Naliri + nivolumab | PD-1 | Ib/II | 2 line or later | 34 | Tolerability PFS |
| ICIs plus tyrosine kinase inhibitor | ||||||
| NCT03797326 | Pembrolizumab + lenvatinib | PD-1 TKI |
II multicohort | Pretreated solid tumors including BTC | 590 | ORR |
| NCT04211168 | Toripalimab + lenvatinib | PD-1 TKI |
II | 2 line or later | 44 | ORR Rate AEs |
| NCT04010071 | Toripalimab + axitinib | PD-1 TKI |
II | 2 line or later | 60 | PFS ORR |
| NCT03475953 | Regorafenib + avelumab | PD-L1 TKI |
i/II | Pretreated solid tumors including BTC | 482 | Recommended phase 2 dose ORR |
| Other combinatory strategies | ||||||
| NCT04057365 | Nivolumab + DKN-01 | PD-1 DKK1 |
II | 2 line or later | 30 | ORR |
| NCT03201458 | Atezolizumab + cobimetinib | PD-L1 MEKi |
II | 2 line or later | 76 | PFS |
| NCT03250273 | Nivolumab + etinostat | PD-1 HDAC |
II | 2 line or later | 44 | ORR |
| NCT04298021 | AZD6738 + durvalumab | PD-1 ATM/ATR |
II | 2 line or later | 74 | DCR |
| NCT03639935 | Nivolumab + rucaparib maintenance after platinum-based chemotherapy |
PD-1 PARP |
II | 1 line | 35 | PFS rate 4 months |
| NCT03991832 | Durvalumab + olaparib | PD-L1 PARP |
II | IDH1 mutated tumors including patients with pretreated BTC |
78 | ORR Overall disease control rate |
ICIs: immune checkpoint inhibitors. OS: overall survival; PFS: progression free survival; ORR: overall response rate; DCR: disease control rate. PD-1: programmed death 1; PD-L1: programmed death ligand 1; BTC: biliary tract cancer; TKI: tyrosine kinase inhibitor; AEs: adverse events; MEKi: MEK inhibitor; HDAC: histone deacetylase. ATM: ataxia-telangiectasia mutation. ATR: ataxia telangiectasia and Rad3-related protein. PARP: poly ADP ribose polymerase. IDH1: isocitrate dehydrogenase.