Table 2.
Ongoing immunotherapy clinical trials targeting the immune microenvironment in cholangiocarcinoma.
| Intervention | Trial type | Population (# participants, estimated enrolment) | ClinicalTrials.gov Identifier |
|---|---|---|---|
| Immune microenvironment targeted therapy | |||
| Interferon alpha + G-CSF + fluorouracil + hydroxyurea | Phase II, single arm, open label | Unresectable GI cancers, including BTC (60 pts) | NCT00019474 |
| INCB001158 (arginase inhibitor) + FOLFOX, GEMCIS or paclitaxel chemotherapy | Phase I/II, non-randomized, open label | Advanced solid tumours including BTC (249 pts) | NCT03314935 |
| Recombinant interleukin-12 + trastuzumab (anti-HER2 antibody) | Phase I, single arm, open label | Advanced, refractory, HER2-expressing solid tumours, including BTC (15 pts) | NCT00004074 |
| CDX-1140 (CD40 agonist antibody) +/- CDX-301 (dendritic cell growth factor) | Phase I, non-randomized, open label | Advanced, refractory, biopsiable cancers, including CCA (180 pts) | NCT03329950 |
| ABBV-368 (OX40 agonist antibody) +/- ABBV-181 (anti-PD-1 antibody) | Phase I, non-randomized, open label | Advanced solid cancers, including CCA (170 pts) | NCT03071757 |
| Immune microenvironment targeted therapy plus immune checkpoint blockade | |||
| Peginterferon alpha-2b + Pembrolizumab | Phase II, single-arm, open label | Advanced, refractory, biopsiable CCA (44 pts) | NCT02982720 |
| Sargramostim (GM-CSF) + Pembrolizumab | Phase II, single arm, open label | Advanced BTC (42 pts) | NCT02703714 |
| Cabiralizumab (anti-CSF1R antibody) + Nivolumab | Phase II, randomized, open label | Resectable, biopsiable BTC (16 pts) | NCT03768531 |
| M7824 (anti-PD-L1/TGFbetaRII fusion protein) | Phase II, single arm, open label | Advanced, refractory BTC (141 pts) | NCT03833661 |
| Entinostat (histone deacetylase inhibitor) + Nivolumab | Phase II, non-randomized, open label | Advanced, untreated CCA or PDAC (54 pts) | NCT03250273 |
| Ramucirumab (anti-VEGFR-2 antibody) + Pembrolizumab | Phase I, single arm, open label | Select advanced, refractory, biopsiable cancers, including BTC (155 pts) | NCT02443324 |
| Lenvatinib (VEGFR2 inhibitor) + Pembrolizumab | Phase II, single arm, open label; Phase II, single arm, open label |
Advanced, refractory, primary liver cancer or BTC (50 pts); Selected advanced, refractory solid tumours, including BTC (180 pts) |
NCT03895970; NCT03797326 |
| Anlotinib hydrochloride (multi-RTK and VEGFR2-3 inhibitor) + TQB2450 (Anti-PD-L1 antibody) | Phase Ib/II, single arm, open label | Advanced, refractory BTC or HCC (60 pts) | NCT03825705 |
| Avelumab (anti-PD-L1 antibody) with Regorafenib (multi-RTK and VEGFR 2/3 inhibitor) | Phase I/II, non-randomized, open label | Advanced, refractory digestive tumours, not MMR-deficient (212 pts) | NCT03475953 |
| pegylated recombinant human hyaluronidase PH20 + atezolizumab + GEMCIS chemotherapy | Phase I, randomized, open label | Advanced, untreated BTC (70 pts) | NCT03267940 |
Ongoing clinical trials were identified by searching ClinicalTrials.gov using the terms "Biliary Cancer," "cholangiocarcinoma," "biliary carcinoma," "bile duct," or "biliary tract" and manually curated for inclusion of an immunotherapy arm. Trials were included with status of "Recruiting," "Not yet recruiting," "Active, not recruiting," "Completed," or "Enrolling by invitation." Trials of general solid tumours were excluded unless a BTC arm or inclusion was specified. Search was updated as of 4/1/19. BTC, biliary tract cancer; GI, gastrointestinal; HCC, hepatocellular carcinoma; iCCA, intrahepatic cholangiocarcinoma; MMR, mismatch repair; PDAC, pancreatic ductal adenocarcinoma.