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. 2023 Jul 8;42(3):1021–1054. doi: 10.1007/s10555-023-10121-2

Table 7.

Ongoing clinical trials of molecular-targeted agents in NEC

Trials Targets Agents Line Phase Primary sites Treatment
mTOR inhibitors
NCT02113800 mTOR Everolimus (Eve) 2nd II Any Mono
NCT02687958 1st II GEP (Ki-67 < 55%) Mono
NCT02248012 1st II GEP or UK (Ki-67 < 55%) Eve + TEM
NCT02695459 1st II Any Eve + CDDP
Anti-angiogenetic inhibitors
NCT04705519 VEGF-A Bevacizumab (Bev) 2nd II Any Bev + nab-PTX
NCT03457844 VEGFR Anlotinib (Anlo) 2nd II Any Mono
NCT05165407 Surufatinib (Sur) 2nd II Any Sur + Sin + IBI310
NCT05015621 2nd III Any Sur + Tori vs. FOLFIRI
NCT04412629 Cabozantinib (Cab) 2nd II GEP Mono
NCT04400474 Any II Any Cab + Atezo
NCT04079712 2nd II Any Cab + Nivo + Ipi
Inhibitors of cell-cycle or DDR regulators
NCT04514497 ATR Elimusertib (Elimu) 2nd II Any Elimu + CPT-11/TPT
NCT04802174 Berzosertib (Berzo) 2nd I/II Any Berzo + Lurbinectedin
NCT02487095 1st II Any (only SCNEC) Berzo + TPT
NCT03896503 1st II Any (only SCNEC) TPT ± Berzo
NCT04209595 PARP Rucaparib (Ruca) 2nd I/II Any (only SCNEC) Ruca + PLX038
NCT04701307 Niraparib (Nira) 2nd II Any Nira + Dostarlimab
Inhibitors of epigenetic regulators
NCT05076786 HDAC Tucidinostat (Tuci) 1st II Extrapulmonary Tuci + CDDP/CBDCA + ETP
NCT05113355 2nd II Any Tuci + Sin
Immune checkpoint inhibitors
NCT03147404 PD-L1 Avelumab 2nd II GEP Mono
NCT05058651 Atezolizumab (Atezo) 1st II/III Any CDDP/CBDCA + ETP ± Atezo
NCT05142865 PD-1 Camrelizumab (Camre) 1st II Extrapulmonary Camre + CDDP/CBDCA + ETP + Apatinib
NCT03992911 Toripalimab (Tori) 1st II/III Any Tori + Simmtecan + 5-FU/LV vs. CDDP/CBDCA + ETP
NCT03517488 PD-1/CTLA-4 XmAb20717 2nd I Any Mono
NCT05337735 2nd II Any Mono
DLL3 targeting agents
NCT04429087 DLL3/CD3 BI 764532 2nd I Any (DDL3 +) Mono
NCT04471727 DLL3/CD3 HPN328 2nd I/II Any (DDL3 +) Mono
SSA/PRRT
NCT02409849 SSTR Octreotide-LAR 1st II GEP Mono
NCT00978211 90Y-/177Lu-Dotatate-TOC Any II Any (SSTR2 +) Mono
NCT04525638 177Lu-Dotatate 1st or 2nd II GEP, lung, or UK Nivo + 177Lu-Dotatate
NCT02936323 PEN-221 2nd I/Iia Any (SSTR2 +) Mono
Others
NCT02250885 XPO1 Selinexor 2nd II GEP, lung, or UK Mono
NCT05126433 RNA polymerase II Lurbinectedin 2nd II Any Mono

Abbreviations: NCT number, ClinicalTrials.gov Identifier; GEP, gastro-entero-pancreatic; NEC, neuroendocrine carcinoma; UK, unknown; Mono, monotherapy; TEM, temozolomide; CDDP, cisplatin; CBDCA, carboplatin; nab-PTX, nanoparticle albumin-bound-paclitaxel; Sin, sintilimab (anti-PD-1 Ab); Nivo, nivolumab (anti-PD-1 Ab); Ipi, ipilimumab (anti-CTLA-4 Ab); CPT-11, irinotecan, TPT, topotecan; ETP, etoposide; 5-FU, 5-fluorouracil; LV, leucovorin; SSA, synthetic somatostatin analog; PRRT, peptide receptor radionuclide therapy; SSTR, somatostatin receptor; LAR, long acting release; XPO, exportin-1; Anlotinib, a tyrosine kinase inhibitor that targets VEGFR, FGFR, PDGFR, and KIT; Surufatinib, a small molecule kinase inhibitor of VEGFR1–3, FGFR1, and CSF-1R; Cabozantinib, a small molecule inhibitor of MET, VEGFR, RET, KIT, and the TAM (TYRO3, AXL, MER) family of receptor kinases; IBI310, anti-CTLA-4 Ab; FOLFIRI, 5-fluorouracil + leucovorin + irinotecan; PLX038, pegylated topoisomerase inhibitor SN-38; Dostarlimab, anti-PD-1 Ab; Apatinib, an inhibitor of VEGFR2; XmAb20717, a humanized bispecific monoclonal antibody of PD-1 and CTLA-4; BI 764532, DLL3/CD3 bispecific T-cell engager; HPN328, DLL3/CD3 bispecific T-cell engager