Table 2.
Molecular target | Study identifier | Study title | Study design | Population | Intervention | Primary endpoint |
---|---|---|---|---|---|---|
Angiogenesis | NCT01841736 | – | Phase 2 RANDOMIZEDDOUBLE-blind | Advanced well differentiated NENs | Pazopanib vs PCB | PFS |
NCT00605566 | – |
Phase 2 Single arm Open-label |
Advanced well differentiated NENs | Sorafenib + metronomic cyclophosphamide | ORR | |
NCT02795858 | – |
Phase 2 Single arm Open-label |
Advanced extra-pancreatic well differentiated NENs | Ramucirumab + SSA | PFS | |
NCT01782443 | – |
Phase 2 Single arm Open-label |
Advanced well differentiated NENs | Ziv-aflibercept | PFS | |
NCT03375320 | CABINET |
Phase 3 Randomized double-blind |
Advanced well differentiated NENs | Cabozantinib vs PCB | PFS | |
NCT02588170 | – |
Phase 3 Randomized double-blind |
Advanced extra-pancreatic well differentiated NENs | Surufatinib vs PCB | PFS | |
NCT02259725 | – |
Phase 2 Single arm Open-label |
Advanced well differentiated NENs | Regorafenib | PFS | |
NCT01744249 | – |
Phase 2–3 Randomized double-blind |
Advanced extra-pancreatic well differentiated NENs | Axitinib + Octreotide LAR vs PCB + Octreotide LAR | PFS | |
NCT02399215 | – |
Phase 2 Single arm Open-label |
Advanced extra-pancreatic well differentiated NENs | Nintedanib | PFS | |
ErbB | NCT00843531 | – | Phase 2 | Moderately to well differentiated advanced NENs | Everolimus + Erlotinib | ORR |
ALK | NCT02568267 | STARTRK-2 |
Phase 2 Basket study |
Solid tumors (including NENs) harboring NTRK 1/2/3, ROS1 or ALK rearrangement | Entrectinib | ORR |
CDK 4/6 | NCT02420691 | – |
Phase 2 Single arm Open-label |
Foregut advanced well differentiated NENs | Ribociclib | ORR |
NCT03070301 | – |
Phase 2 Single arm Open-label |
Foregut advanced well differentiated NENs | Ribociclib + Everolimus | PFS | |
Immuno-therapy | NCT02955069 | – |
Phase 2 Single arm Open-label |
Advanced well differentiated NENs of pancreatic, GI or thoracic origin and advanced GEP-NECs | PDR-001 | ORR |
NCT03278379 | NET-002 |
Phase 2 Single arm Open-label |
Advanced well differentiated NENs |
Avelumab | ORR | |
NCT03420521 | – |
Phase 2 Single arm Open-label |
Advanced well differentiated NENs of pancreatic, GI or lung origin (3 cohorts) | Nivolumab + Ipilimumab | ORR | |
NCT02923934 | – |
Phase 2 Single arm Open-label |
Rare cancers (including NENs) | Nivolumab + Ipilimumab | CBR | |
NCT03095274 | DUNE |
Phase 2 Single arm Open-label |
Advanced well differentiated NENs of pancreatic, GI or lung origin and advanced GEP-NECs (4 cohorts) | Durvalumab + Tremelimumab | CBR | |
NCT03728361 | – |
Phase 2 Single arm Open-label |
Progressive SCLC and advanced NENs (2 cohorts) | Nivolumab + Temozolomide | ORR |
PCB placebo, PFS progression free survival, ORR objective response rate, SSAs somatostatin analogs, ORR objective response rate, GI gastrointestinal, GEP-NECs gastroenteropancreatic neuroendocrine carcinomas, CBR clinical benefit rate, SCLC small cell lung cancer