Novel agents in clinical trials for treating patients with MDS
| Agent . | MOA . | Patient diagnosis . | Clinicaltrials.gov No. . |
|---|---|---|---|
| Improving HMT delivery . | . | . | |
| SGI-110 | Oral HMT: novel dinucleotide of DAC + deoxyguanosine resistant to degradation by CDA | MDS and AML | NCT01261312, plus others |
| CC-486 | Oral HMT: azacytidine | MDS | NCT02281084, plus others |
| ASTX727 | Oral CDAi (E7727) + oral DAC | MDS | NCT02103478 |
| Mocetinostat MGCD0103 | Class 1 HDACi | AML and MDS | NCT02018926, plus others |
| 4SC-202 | Class 1 HDACi and LSD-1 inhibitor | MDS and hematologic cancers | NCT01344707 |
| Panbinostat | Class 1 and 2 HDACi alone + HMT and others | AML and MDS | NCT01451268, plus others |
| New epigenetic targets | |||
| AG-120 | IDH1 inhibitor | MDS/AML with IDH1 mutation | NCT02074839 |
| AG-221 | IDH2 inhibitor | MDS/AML with IDH2 mutation | NCT01915498 plus others |
| CPI613 | PDH inhibitor | AML and MDS | NCT01902381 |
| CPI-0610, OTX015 | BET inhibitor | AML, MDS, and MPN | NCT02158858 |
| Immune-based approach | |||
| Nivolumab | PD1 inhibitor | MDS/AML | NCT02464657/NCT01822509 |
| Ipilumumab (MDX-101) | CTLA-4 inhibitor | MDS/AML | NCT01757639/NCT01822509 |
| Durvalumab (MEDI4736) | PD-L1 inhibitor (± tremelimumab) with or without 5AC | MDS/AML | NCT02117219 and others |
| Inhibit signal transduction | |||
| ARRY 614 | Dual inhibitor of P38 MAPK and Tie2 | MDS | NCT01496495 |
| Volasertib | PLK-1 inhibitor | MDS | NCT01241500 and others |
| Rigosertib | Multikinase inhibitor | MDS | NCT02562443 plus others |
| Erlotinib | EGFR inhibitor | MDS | NCT00977548 |
| Other | |||
| Tosedostat | Aminopeptidase inhibitor | MDS/AML | NCT02452346 |
| Vosaroxin SNS-595 | Quinolone derivative | Higher risk MDS | NCT01980056 and others |
| Agent . | MOA . | Patient diagnosis . | Clinicaltrials.gov No. . |
|---|---|---|---|
| Improving HMT delivery . | . | . | |
| SGI-110 | Oral HMT: novel dinucleotide of DAC + deoxyguanosine resistant to degradation by CDA | MDS and AML | NCT01261312, plus others |
| CC-486 | Oral HMT: azacytidine | MDS | NCT02281084, plus others |
| ASTX727 | Oral CDAi (E7727) + oral DAC | MDS | NCT02103478 |
| Mocetinostat MGCD0103 | Class 1 HDACi | AML and MDS | NCT02018926, plus others |
| 4SC-202 | Class 1 HDACi and LSD-1 inhibitor | MDS and hematologic cancers | NCT01344707 |
| Panbinostat | Class 1 and 2 HDACi alone + HMT and others | AML and MDS | NCT01451268, plus others |
| New epigenetic targets | |||
| AG-120 | IDH1 inhibitor | MDS/AML with IDH1 mutation | NCT02074839 |
| AG-221 | IDH2 inhibitor | MDS/AML with IDH2 mutation | NCT01915498 plus others |
| CPI613 | PDH inhibitor | AML and MDS | NCT01902381 |
| CPI-0610, OTX015 | BET inhibitor | AML, MDS, and MPN | NCT02158858 |
| Immune-based approach | |||
| Nivolumab | PD1 inhibitor | MDS/AML | NCT02464657/NCT01822509 |
| Ipilumumab (MDX-101) | CTLA-4 inhibitor | MDS/AML | NCT01757639/NCT01822509 |
| Durvalumab (MEDI4736) | PD-L1 inhibitor (± tremelimumab) with or without 5AC | MDS/AML | NCT02117219 and others |
| Inhibit signal transduction | |||
| ARRY 614 | Dual inhibitor of P38 MAPK and Tie2 | MDS | NCT01496495 |
| Volasertib | PLK-1 inhibitor | MDS | NCT01241500 and others |
| Rigosertib | Multikinase inhibitor | MDS | NCT02562443 plus others |
| Erlotinib | EGFR inhibitor | MDS | NCT00977548 |
| Other | |||
| Tosedostat | Aminopeptidase inhibitor | MDS/AML | NCT02452346 |
| Vosaroxin SNS-595 | Quinolone derivative | Higher risk MDS | NCT01980056 and others |
CDAi, CDA inhibitor; CTLA-4, T-lymphocyte–associated antigen 4; EGFR, epidermal growth factor receptor; LSD-1, lysine-specific demethylase 1; MOA, mechanism of action; MPN, myeloproliferative neoplasm; PDH, pyruvate dehydrogenase; PLK-1, polo-like kinase 1.