Table 4.
Selected list of investigational agents.
| Agent | Mechanism of action | Phase of investigation | ClinicalTrials.gov identifier | Reference |
|---|---|---|---|---|
| Drugs that target the microenvironment or leukemic stem cell | ||||
| PF-04449913 | Hedgehog inhibitor | I/II | NCT01546038 | (56) |
| Kinase inhibitors | ||||
| Volasertib | PLK1 inhibitor | III | NCT01721876 | (57, 58) |
| GSK2141795 + trametinib | RAS activation pathway inhibitor | II | NCT01907815 | (59) |
| Quizartinib | FLT3 inhibitor | I/II | NCT01892371 | (60, 61) |
| ASP2215 | FLT3/AXL inhibitor | I | NCT02014558 | (62) |
| Pevonedistat | NAE inhibitor | I | NCT01814826 | (63, 64) |
| Epigenetic therapies | ||||
| Pracinostat | HDAC inhibitor | II | NCT01912274 | (65) |
| Valproic acid | HDAC inhibitor | II | NCT00867672 | (66, 67) |
| NCT00414310 | ||||
| Vosaroxin | Topoisomerase II inhibitor | I/II | NCT01893320 | (68) |
| Bortezomib | NF-kB pathway inhibitor | II | NCT01420926 | (36) |
| AG-120 | IDH1 | I | NCT02074839 | (69) |
| AG-221 | IDH2 | I | NCT01915498 | (70) |
| EPZ-5676 | DOT1L | I | NCT02141828 | (71) |
| SGI-110 | DNA hypomethylator | I–II | NCT01261312 | (72) |
| Others | ||||
| Selinexor | SINE | II | NCT02088541 | (73) |
SINE, selective inhibitor of nuclear export.