Table 3

New therapies under evaluation in biliary tract cancers

Therapeutic regimenTargetPhaseClinicalTrials.gov identifierStatus
VEGF and multitarget
RamucirumabVEGFR22NCT02520141Recruiting
GEMCIS and ramucirumab or merestinibVEGFR2
c-MET
2NCT02711553Recruiting
GEMOX and sorafenibVEGFR-2/3, PDGFR-β, B-Raf, C-Raf1/2NCT00955721Completed (efficacy not reported)
GEM and sorafenibVEGFR-2/3, PDGFR-β, B-Raf, C-Raf
FOLFOX and cediranibVEGFR2NCT01229111Not recruiting (no results)
RegorafenibVEGFR1-3, c-KIT, TIE-2, PDGFR-β, C-Raf, B-Raf, p38 MAPK, FGFR1-2, Ret2
2
NCT02115542
NCT02053376
Recruiting
Recruiting
MAPK
Trametinib versus CAP/5 FUMEK 1/22NCT02042443Ongoing (not recruiting)
Binimetinib (MEK162) and GEMCISMEK 1/22NCT01828034Active (not recruiting)
Binimetinib and GEMOXMEK 1/21 NCT02105350 Suspended (no results)
Binimetinib (MEK162) and capecitabineMEK1NCT02773459Recruiting
Selumetinib (at different doses) and GEMCISMEK 1/22NCT02151084Recruiting
MEK162 and capecitabineMEK1NCT02773459Recruiting
mTOR
EverolimusmTOR2NCT00973713Unknown (not verified on ClinicalTrials.gov)
GEMCIS and everolimusmTOR1NCT00949949Not recruiting (no results)
Other
ARG 087FGFR1/2NCT01752920Recruiting
Cabozantinibc-MET, VEGFR22NCT01954745Active, not recruiting
AG-221IDH2 mutation1/2NCT02273739Completed (not reported)
GEMCIS and ADH1 (Exherin)N-cadherin1NCT01825603Recruiting
Immunotherapy
NivolumabPD12NCT02829918Recruiting
Pembrolizumab and GM-CSFPD12NCT02703714Recruiting
Ipilimumab and nivolumabCTLA4 and PD12NCT02923934Not yet recruiting
Dendritic cell-precision T cells for neoantigen (DC-PNAT) and gemcitabinePersonalised neoantigens2NCT02632019
Multiagent
Multiple arms based on molecular profiling (cetuximab, trastuzumab, gefitinib, lapatinib, everolimus, sorafenib, crizotinib)Multiple (EGFR, HER2, mTOR, VEGF/EGFR/PDGFR, ALK/ROS1)2NCT02836847Recruiting
Ramucirumab and pembrolizumab1NCT02443324Recruiting
  • All cited from ClinicalTrials.gov on 21 November 2016.

  • 5 FU, 5-fluorouracil; ALK, anaplastic lymphoma kinase; BRAF, v-Raf murine sarcoma viral oncogene homologue B; CAP, capecitabine; CIS, cisplatin; C-Raf, RAF proto-oncogene serine/threonine protein kinase; CTLA4, cytotoxic T-lymphocyte associated protein 4; EGFR, epidermal growth factor receptor; FGFR, fibroblast growth factor receptor; FOLFOX, 5-fluorouracil and oxaliplatin; GEM, gemcitabine; GM-CSF, granylocyte macrophage colony stimulating factor; HER2, human epidermal growth factor receptor 2; IDH, isocitrate dehydrogenase; MAPK, mitogen-activated protein kinase; mTOR, mammalian target of rapamycin; OX, oxaliplatin; PDGFR, platelet-derived growth factor receptor; PD1, programmed cell death protein 1; ROS1, c-ros oncogene 1; VEGF, vascular endothelial growth factor; VEGFR, vascular endothelial growth factor receptor.