Table 2.
Ongoing clinical trials evaluating the effects of multitarget tyrosine kinase inhibitors, acting also on FGFR, in patients with NENs.
| Identifier | Therapy | Molecular target | Study design | Tumors | Estimated sample size | Primary outcome | Start date | Estimated Completion Date |
|---|---|---|---|---|---|---|---|---|
| NCT02399215 | Nindetanib | FGFR VEGFR PDGFR |
Multicenter open label phase II study | Well or moderately differentiated (G1, G2) NEN not pancreatic | 30 | PFS | May 2015 | October 2020 |
| NCT04207463 | Anlotinib + AK105 (anti PD1) |
FGFR VEGFR PDGFR c-kit |
Multicenter multi-cohort open label phase II study |
G1 or G2 GEP NET (cohort 5) | 150 (all cohorts) | ORR | June 2020 | December 2020 |
| NCT02259725 | Regorafenib | FGFR VEGFR1-3 TIE2 KIT RET RAF-1 BRAF BRAFV600E PDGFR |
Multicenter multi-cohort open-label phase II study |
Carcinoid (cohort A) or pancreatic islet cell tumors (cohort B) | 48 | PFS | August 2016 | August 2021 |
| NCT03950609 | Lenvatinib + Everolimus (mTOR inhibitor) | FGFR1-4 VEGFR1-3 | Single center open-label phase II study |
Unresectable well differentiated carcinoid tumors |
32 | ORR | July 2019 | May 2021 |
| NCT03475953 | Regorafenib + Avelumab (anti PD-L1) | FGFR VEGFR1-3 TIE2 KIT RET RAF-1 BRAF BRAFV600E PDGFR |
Multicenter, open label phase I/II study | G2 or G3 GEP NEN (cohort G) |
362 | ORR (Phase 2) | May 2018 | November 2020 |
| NCT02657551 | Regorafenib | FGFR VEGFR1-3 TIE2 KIT RET RAF-1 BRAF BRAFV600E PDGFR |
Open-label phase II study | Metastatic medullary thyroid cancer | 33 | PFS | January 2016 | October 2022 |
| NCT03008369 | Lenvatinib | FGFR1-4VEGFR1-3 | Open-label phase II study | Metastatic PPGLs | 25 | TRR | May 2017 | December 2020 |
FGFR, fibroblast growth factor receptor; GEP, gastro-entero-pancreatic; NA, not available; NEN, neuroendocrine neoplasm; NET, neuroendocrine tumor; ORR, objective response rate; PD1, Programmed cell death protein 1; PDGFR, Platelet-derived growth factor receptor; PD-L1, Programmed cell death ligand 1; PFS, progression free survival; PPGL, Pheochromocytoma and Paraganglioma; pts, patients; TRR, tumor response rate (complete response and partial response); VEGFR, Vascular Endothelial Growth Factor Receptor.