Skip to main content
. Author manuscript; available in PMC: 2020 Sep 2.
Published in final edited form as: Drugs. 2020 May;80(7):647–669. doi: 10.1007/s40265-020-01304-0

Table 1.

Ongoing Clinical Trials Evaluating Selected Targets/Pathways in Advanced Pancreas Cancer.

Target Population Drug Additional therapy Treatment setting Ongoing clinical trials Status¥
Targeting the KRAS Pathway
HER2 Advanced pancreatic, biliary cancers Afatinib Gemcitabine Any line Phase 1b NCT02451553 Recruiting
Solid tumors with NRG1 fusion Zenocutuzumab None Any line Phase 1/2 NCT02912949 Recruiting
NTRK fusion NTRK/ROS1/ALK gene rearranged advanced/metastatic solid tumors Entrectinib None Any line Phase 2 NCT02568267 Recruiting
ALK, ROS1 gene translocations ALK, ROS1 translocated solid tumors Crizotinib None Any line Phase 2 NCT02465060 (MATCH screening trial) Recruiting
BRAF V600E mutation BRAF V600E/R/K/D mutant solid tumors Dabrafenib Trametinib Any line Phase 2 NCT02465060 (MATCH screening trial) Recruiting
KRASG12C KRASG12C-mutant PDAC, solid tumors AMG-510 Alone or +MEK-inhibitor ND Phase 1b NCT04185883 Recruiting
Alone ND Phase 1/2 NCT03600883 Recruiting
MRTX849 Alone ND Phase 1/2 NCT03785249
Autophagy KRAS-mutant PDAC Hydroxychloroquine GA Any Phase 1/2 NCT01506973 Recruiting
MEK-inhibitors: Binimetinib, Trametinib Any Phase 1 NCT03825289, NCT04132505 Recruiting
KRASG12D KRASG12D-mutant mPDAC KRASG12D siRNA None Second-line or more Phase 1 NCT03608631 Recruiting
Tumor Metabolism
Mitochondrial metabolism mPDAC Devimistat (CPI-613) (m)FOLFIRINOX First-line Phase 3 NCT03504423 Recruiting
Ubidecarenone (BMP31150) Gemcitabine Second- or third-line Phase 2 NCT02650804 Recruiting
Eryaspase GA or 5-FU/LV plus Irinotecan Second-line Phase 3 NCT03665441 Recruiting
Targeting the Tumor Microenvironment
Focal adhesion kinase (FAK) Advanced solid tumors
Advanced PDAC		 Defactinib Gemcitabine, pembrolizumab Second-line Phase 1 NCT02546531 Recruiting complete
Pembrolizumab Exhausted standard CT Phase 1/2 NCT02758587
GSK2256098 Trametinib Exhausted standard CT Phase 2 NCT02428270 Complete
Vitamin D pathway mPDAC Paricalcitol Pembrolizumab Maintenance Phase 2 NCT02428270 Phase 2 NCT03331562, NCT03520790 Complete
IDO1 inhibition mPDAC Epacadostat Pembrolizumab, CRS-207 Second-line Phase 2 NCT03006302 Recruiting
Immunotherapeutic strategies: Beyond MMR Gene Defects, MSI-H tumors
PD-1 mPDAC, advanced PDAC Pembrolizumab PEGPH20 Second-line Phase 2 NCT04058964 Not yet recruiting
Paricalcitol Maintenance Phase 2 NCT03331562 Recruiting
Azacytidine Second-line Phase 2 NCT03264404 Recruiting
BL-8040 Second-line Phase 2b NCT02907099 Complete
Sonidegib Second-line Phase 1 (Part B) NCT04007744 Recruiting
Nivolumab Cabiralizumab+ gemcitabine/nab-paclitaxel Second-line Phase 2 NCT03697564 Recruiting
PD-L1 RAS-mutant solid tumors (mPDAC) Avelumab Binimetinib+ talazoparib Second-line or later Phase 1b/2 NCT03637491 Recruiting
Oncolytic virus Advanced PDAC Pelareorep Pembrolizumab Second-line Phase 2 NCT03723915 Recruiting
Peptide vaccine mPDAC, CRC Tumor associated peptide vaccine Personolized peptide vaccine Imiquimod (topical) Pembrolizumab Pilot NCT02600949 Active not recruiting
T cells therapy Metastatic GI cancers Autologous CD 8+ T cells Pembrolizumab Second-line or more Pilot NCT02757391 Complete
CD40 mPDAC APX005M (CD40 agonist monoclonal Ab) GA± nivolumab First-line Phase 1b/2 NCT03214250 Active not recruiting
Combination immunotherapy mPDAC CRS-207, nivolumab, ipilimumab ± GVAX Second-line Phase 2 NCT03190265 Recruiting
Epacadostat Pembrolizumab, CRS-207 Second-line Phase 2 NCT03006302 Recruiting
Atezolizumab CT, multiple immunotherapy (Emactuzumab, Selicrulumab, etc.) Cohort 1: First-line; Cohort 2: second line Phase 1b/2 NCT03193190 Recruiting
mPDAC Durvalumab+ tremelimumab GA First-line Phase 2 NCT02879318 Recruiting
DDR Gene Mutation
DDR gene Germline/somatic BRCA, PALB2 mutated mPDAC (not progressed on platinum-based therapy) Rucaparib None Maintenance Phase 2 NCT03140670 Recruiting
Germline/somatic DDR gene mutated solid tumors Rucaparib None Second-line Phase 2 NCT04171700 Recruiting
mPDAC (other GI cancers) with DDR gene mutations Rucaparib 5-FU/LV + Irinotecan First-line Phase 2 NCT03337087 Recruiting
mPDAC (±DDR gene mutation) Rucaparib FOLFIRI Second-line Phase 2 NCT02890355 Active, not recruiting
Advanced PDAC with BRCA1/2, PALB2, CHEK2 or ATM mutation Niraparib None Second-line or more Phase 2 NCT03601923 Recruiting
Phase 2 NCT03553004 Recruiting
mPDAC with DDR gene mutations Niraparib Nivolumab or ipilumumab (Parpvax) Second-line or more Phase 1b/2 NCT03404960 Recruiting
Other Approaches
Adenosine Advanced solid cancers Ciforadenant (A2AR antagonist) CPI006, pembrolizumab Second-line or more Phase 1/1b NCT03454451 Recruiting
NIR178 (A2AR antagonist) PDR001 (anti-PD-1) Second-line or more Phase 2 (nonrandomized) NCT03207867 Recruiting
TTX-030 (anti-CD39 antibody Pembrolizumab or chemotherapy ND Phase 1/1b (nonrandomized) NCT03884556 Recruiting
AXL pathway mPDAC Bemcentinib (BGB324) GA First-line Phase 1b/2 NCT03649321 Recruiting
RNAi Locally advanced PDAC siG12D-LODERTM GA First-line Phase 2 NCT01676259† Recruiting
PDGFR mPDAC Olaratumab GA First-line Phase 1b/2 NCT03086369
Hedgehog pathway Advanced solid tumors Sonidegib Pembrolizumab Phase 1 NCT04007744 Not yet recruiting
Toll-like receptor Refractory mPDAC SD-101 Nivolumab, radiation Second-line or more Pilot NCT04050085
¥

Date accessed 02/02/2020; § Trials of PEGPH20 has been negative so far;

Also includes phase 1 and phase 1b.

Δ

Part 1 and part 2;

†

Being amended to include (m)FOLFIRINOX.

mPDAC metastatic pancreatic ductal adenocarcinoma; GA gemcitabine plus nab-paclitaxel; mFOLFIRINOX modified regimen of 5-fluorouracil, leucovorin, irinotecan, and oxaliplatin; 5-FU/LV 5-fluorouracil plus leucovorin; HA hyaluronic acid; CT chemotherapy; IDO1 indoleamine 2,3-dioxygenase-1; CRS-207 Listeria monocytogenes expressing mesothelin; CRC colorectal cancer; GI gastrointestinal; ND not defined.