Table 5.
Current trials involving molecular treatments in BTC registered on clinicaltrials.gov
| NCT; references | Study | Phase | Status | Recruiting countries | Comments |
|---|---|---|---|---|---|
| NCT03144856152 | Apatinib as second-line treatment in aBTC | II | Recruiting | China | VEGFR2 TKI |
| NCT02579616153 | Lenvatinib (E7080) in unresectable BTC who have failed Gem-based combination | II | Active, not recruiting | Japan | VEGFR1/2/3 TKI |
| NCT02773459154 | To test the efficacy of MEK162 with Cape in Gem-pretreated aBTC, predict biomarkers | I/II | Recruiting | Korea | MEK inhibitor |
| NCT03093870155 | Multicentre double-blind, placebo-controlled study of varlitinib plus capecitabine vs placebo plus capecitabine in aBTC or mBTC as second-line treatment | II/III | Not yet recruiting | USA | EGFR, HER2, HER4 inhibitor |
| NCT03129074156 | Varlitinib and Cape in aBTC and mBTC patients | II | Not yet recruiting | NA | EGFR, HER2, HER4 inhibitor |
| NCT03110484157 | Pemetrexed with erlotinib as salvage treatment in mBTC patients who have failed Gem | II | Not yet recruiting | Korea | EGFR TKI |
| NCT02992340158 | Varlitinib with GC for treatment-naïve aBTC or mBTC | I/II | Recruiting | Korea, Singapore, Taiwan | EGFR, HER2, HER4 inhibitor |
| NCT02711553159 | Ramucirumab or merestinib or placebo with GC in aBTC or mBTC | II | Recruiting | Multinational | Ramucirumab = VEGFR2 antibody Merestinib = MET inhibitor |
| NCT02966821160 | Sulfatinib as second-line treatment in patients with BTC | II | Recruiting | China | VEGFR and FGFR1 TKI |
| NCT02520141161 | Ramucirumab for pretreated aBTC | II | Recruiting | USA | VEGFR2 antibody |
| NCT02836847162 | Molecularly targeted therapy with GemOx in aEHC or rEHC and GBC | II | Recruiting | China | Precision target therapy |
| NCT02443324163 | Ramucirumab with pembrolizumab in gastric, GEJ adenoca, NSCLC, TCC in urothelium or BTC | I | Recruiting | Multinational | Ramucirumab = VEGFR2 antibody Pembrolizumab = PD-1 antibody |
| NCT02386397164 | Regorafenib dose for Phase II trial in combination with mGemOx in aBTC | I/II | Recruiting | France | Dual VEGFR-TIE2 TKI |
| NCT01825603165 | ADH-1 with GC in patients with irresectable BTC/pancreatic ca | I | Recruiting | USA | Alcohol dehydrogenase 1 blocks N-cadherin |
| NCT03082053166 | Varlitinib in Japanese subjects with advanced or MET solid tumors | I | Recruiting | Japan | EGFR, HER2, HER4 inhibitor |
| NCT00948935167 | Panitumumab with GemIr in aBTC and mBTC | II | Ongoing but not recruiting | USA | EGFR antibody |
| NCT0215108448 | Different dosing schedules of selumetinib with GC vs GC alone in BTC | II | Recruiting | Canada | MEK inhibitor |
| NCT02265341168 | Ponatinib in aBTC patients with FGFR2 fusions | II | Recruiting | US | BCR–ABL inhibitor |
| NCT02451553169 | Afatinib with Cape in advanced refractory solid tumors, pancreatic ca, BTC | I | Recruiting | USA | EGFR TKI |
| NCT02943031170 | Effect of individualized precision therapy programs in patients with BTC | IV | Not yet recruiting | China | Precision therapy |
| NCT03185988171 | Patients with metastatic disease of the digestive system | II | Not yet recruiting | China | Anti-HER2 therapy |
| NCT02042443172 | Trametinib or combination chemotherapy in patients with refractory or advanced BTC or GBC that is irresectable | II | Active, not recruiting | USA | MEK inhibitor |
| NCT02586987173 | Study to assess the safety and tolerability of ascending doses of selumetinib with MEDI4736 and selumetinib, MEDI4736, tremelimumab in advanced solid tumors | I | Recruiting | Multinational | Selumetinib = MEK inhibitor MEDI4736 = PD-L1 antibody Tremelimumab = CTLA4 antibody |
| NCT02128282174 | CX-4945 in combination with GC as frontline treatment for CCA | I/II | Recruiting | Multinational | CK2 protein kinase inhibitor |
| NCT02053376175 | Phase II trial of regorafenib in aBTC and mBTC, CCA patients who have failed first-line chemotherapy | II | Recruiting | USA | Dual VEGFR-TIE2 TKI |
| NCT02715089176 | Precise treatment in hepatobiliary cancers | Observa- tional | Recruiting | China | Precision treatment |
| NCT01752920177 | Phase I/II study of ARQ087 in advanced solid tumors with FGFR genetic alterations | I/II | Active, not recruiting | USA | Pan-FGFR inhibitor |
| NCT01855724178 | Clinical trial to investigate the efficacy of treatment with Gem and pazopanib in BTC | II | Recruiting | Greece | C-KIT, FGFR, PDGFR and VEGFR inhibitor |
| NCT02631590179 | Copanlisib with GC in aCCA | II | Recruiting | USA | PI3K inhibitor |
| NCT02576431180 | Study of LOXO-101 (larotrectinib) in subjects with NTRK fusion-positive solid tumors (NAVIGATE) | II | Recruiting | Multinational | Tropomyosin receptor kinase inhibitor |
| NCT03027284181 | Merestinib in Japanese patients with advanced or metastatic ca | I | Recruiting | Japan | MET inhibitor |
| NCT01766219182 | CPI-613 in irresectable, advanced or metastatic BTC | I/II | Recruiting | USA | PDH and αKGDH inhibitor |
| NCT02495896183 | Recombinant EphB4-HSA fusion protein with standard chemo in advanced or metastatic solid tumors | I | Recruiting | USA | Recombinant fusion protein composed of full length extracellular domain soluble of human receptor TK ephrin type B receptor 4 |
| NCT0363993591 | Rucaparib in combination with nivolumab in patients with advanced or mBTC following platinum | II | Not yet recruiting | USA | PARP inhibitor and PD-1 antibody |
| NCT02433639184 | Study of TH-302 monotherapy as second-line treatment in aBTC | II | Unknown | South Korea | Hypoxia activated prodrug |
| NCT03185988185 | Anti-HER2 therapy in patients with metastatic disease and HER2-positive disease of the digestive system | II | Not yet recruiting | China | HER2 antibody |
| NCT02115542186 | Single-agent regorafenib in refractory aBTC | II | Active | USA | Multikinase inhibitor |
| NCT03093870187 | Varlitinib in combination with capecitabine for metastatic and aBTC | II/III | Recruiting | Multinational | HER inhibitor |
| NCT0333708791 | Liposomal irinotecan, fluorouracil, leucovorin calcium and rucaparib in treating patients with metastatic pancreatic, colorectal, gastroesophageal or BTC | I/II | Not yet recruiting | USA | PARP inhibitor |
| NCT02715089188 | Precise treatment in hepatobiliary cancers | Recruiting | China | Use of NGS to obtain genomic data and targeting specific mutations with precision medicines | |
| NCT02631590189 | Copanlisib (BAY 80-6946) in combination with GC in aCCA | II | Recruiting | USA | PI3K inhibitor |
| NCT03230318177 | ARQ087 in subjects with FGFR2 gene fusion- positive inoperable or advanced IHC | II | Recruiting | USA, Canada and Italy | Pan-FGFR inhibitor |
Abbreviations: aBTC, advanced biliary tract cancer; aCCA, advanced cholangiocarcinoma; αKGDH, α-ketoglutarate dehydrogenase; BTC, biliary tract cancer; Bcr-abl, bcr-abl fusion oncogene/Philadelphia chromosome; ca, carcinoma; chemo, chemotherapy; CCA, cholangiocarcinoma; cKIT, proto-oncogene c-Kit; CTLA-4, cytotoxic T-lymphocyte-associated protein 4; FGFR, fibroblast growth factor receptor; GEJ, gastro-esophageal junction; GBC, gallbladder cancer; GC, gemcitabine and cisplatin; gem, gemcitabine; GemCape, gemcitabine and capecitabine; GemOx, gemcitabine and oxaliplatin; HCC, hepatocellular carcinoma; HER, human EGF receptor; HER2, human EGF receptor 2; IHC, intrahepatic cholangiocarcinoma; mBTC, metastatic biliary tract cancer; MEK, mitogen-activated protein kinase; MET, MET proto-oncogene, mesenchymal to epithelial transition proto-oncogene; NGS, next generation sequencing; ; NA, not available; NSCLC, non-small-cell lung cancer; PARP, poly-ADP ribose polymerase; PD-1, programmed death 1; PDGFR, plasma-derived growth factor receptor; PDH, pyruvate dehydrogenase; PD-L1, programmed cell death ligand-1; PI3K, phosphoinositide 3-kinase; TIE2, TCC, transitional cell carcinoma; TIE family of angiopoietin receptor kinase; TKI, tyrosine kinase inhibitor; VEGF, vascular endothelial growth factor; VEGFR, vascular endothelial growth factor receptor.