Table 3.
New target therapies under evaluation in clinical trials in iCCA*
| Drug | Phase | Target | Trial enrichment | Primary endpoint |
|---|---|---|---|---|
| Elumetinib | II | MEK1/2 | No | DCR |
| Everolimus | II | mTOR | No | DCR |
| MK2206 | II | AKT | No | ORR |
| AG-120 | I | IDH1 | IDH1 mutation | MTD |
| AG-221 | I/II | IDH2 | IDH2 mutation | MTD, TTP |
| BGJ398 | II | FGFR 1-4 | FGFR alterations | ORR |
| ARQ 087 | I/II | FGFR 1-4 | FGFR alterations | Safety, TTP |
| Regorafenib | II | VEGFR1-3, TIE-2, MAPK, FGFR-1 | No | OS |
| Crizotinib | II | ALK, ROS1, MET | ROS1 or ALK over-expression/genetic alterations | DCR |
| Cabozantinib | II | c-MET, VEGFR2 | No | PFS |
*
The data were accessed in November 2015 on the ClinicalTrials.gov online database.
Abbreviations: DCR, disease control rate; ORR, overall response rate; MTD, maximum-tolerated dose; TTP, time to progression; OS, overall survival; PFS, progression free survival.