Recent or ongoing trials of epigenetic-directed therapies
| Class . | Agent . | Phase . | Target population . | Status . | Identifier . |
|---|---|---|---|---|---|
| HMA | Guadecitabine | 3 | R/R AML | Active, not recruiting | NCT02920008 |
| HMA + DLI | Guadecitabine + DLI | 2 | AML or MDS relapsed post-AlloSCT | Recruiting | NCT02684162 |
| HMA | Guadecitabine | 3 | Newly diagnosed AML ineligible for intensive chemotherapy | Completed | NCT02348489 |
| HMA ± chemotherapy | Guadecitabine ± idarubicin | 2 | AML age ≥70 y | Active, not recruiting | NCT02096055 |
| HMA | Guadecitabine | 2 | AML with 20%-30% blasts or MDS refractory to azacitidine | Completed | NCT02197676 |
| HMA | Guadecitabine | 1/2 | AML, MDS | Completed | NCT01261312 |
| HMA + anti–PD-1 | Guadecitabine + atezolizumab | 1 | AML | Suspended | NCT02892318 |
| HDACi | Entinostat | 2 | AML age ≥60 y | Recruiting | NCT01305499 |
| HDACi + HMA | Entinostat + azacitidine | 1 | AML, MDS, CML | Active, not recruiting | NCT00101179 |
| HDACi + chemotherapy | Panobinostat + cytarabine and daunorubicin | 1 | AML, MDS age ≥60 | Active, not recruiting | NCT01463046 |
| HDACi + chemotherapy | Panobinostat + cytarabine and idarubicin | 1/2 | AML age ≥65 y | Completed | NCT00840346 |
| HDACi + chemotherapy | Panobinostat + cytarabine and idarubicin | 1 | AML age <65 y | Completed | NCT01242774 |
| HDACi + HMA | Panobinostat + azacitidine | 1 | AML <30% blasts, MDL, CMML, | Active, not recruiting | NCT00946647 |
| HDACi + HMA | Panobinostat + decitabine | 1/2 | AML age ≥60, MDS | Completed | NCT00691938 |
| HDACi + HMA | AR-42 + decitabine | 1 | AML | Completed | NCT01798901 |
| HDACi | Belinostat | 2 | AML age ≥60 | Completed | NCT00357032 |
| HDACi + HMA | Mocetinostat + azacitidine | 1/2 | AML, MDS | Completed | NCT00324220 |
| HDACi | Romidepsin | 2 | R/R AML | Completed | NCT00062075 |
| mIDH1 inhibitor | BAY1436032 | 1 | mIDH1 AML | Completed | NCT03127735 |
| mIDH1 inhibitor | Ivosidenib + venetoclax | 1/2 | mIDH1 AML, MDS/MPN | Recruiting | NCT03471260 |
| mIDH1 inhibitor ± HMA, chemotherapy | FT-2102 ± azacitidine or cytarabine | 1/2 | mIDH1 AML, MDS | Recruiting | NCT02719574 |
| mIDH2 inhibitor + HMA | Enasidenib + azacitidine | 2 | R/R mIDH2 AML | Recruiting | NCT03683433 |
| mIDH1, mIDH2 inhibitors + chemotherapy | Ivosidenib or enasidenib + cytarabine and daunorubicin or idarubicin | 1 | Newly diagnosed mIDH1 or mIDH2 AML | Active, not recruiting | NCT02632708 |
| mIDH1, mIDH2 inhibitors + HMA | Ivosidenib or enasidenib + azacitidine | 1/2 | Newly diagnosed mIDH1 or mIDH2 AML ineligible for intensive chemotherapy | Active, not recruiting | NCT02677922 |
| mIDH1 inhibitor + HMA | Ivosidenib or placebo + azacitidine | 3 | Newly diagnosed mIDH1 AML | Recruiting | NCT03173248 |
| BETi | OTX015 (MK-8628) | 1 | AML, ALL, NHL, MM | Completed | NCT01713582 |
| BETi ± HMA | FT-1101 ± azacitidine | 1 | R/R AML | Recruiting | NCT02543879 |
| BETi | GSK525762 | 1 | AML, NHL, MM | Recruiting | NCT01943851 |
| BETi | INCB054329 | 1/2 | AML, MDS/MPN, MM, solid tumor, or lymphoma | Completed | NCT02431260 |
| BETi ± BCL2 inhibitor | ABBV-075 +/− venetoclax | 1 | AML, MM, solid tumors, NHL | Active, not recruiting | NCT02391480 |
| EZH 1/2 inhibitor | DS-3201b | 1 | AML or ALL | Recruiting | NCT03110354 |
| BETi | RO6870810 | 1 | R/R AML and MDS | Completed | NCT02308761 |
| BETi | OTX015 + azacitidine | 1b/2 | Newly diagnosed AML ineligible for intensive chemotherapy | Withdrawn | NCT02303782 |
| LSD1 inhibitor | GSK2879552 | 1 | R/R AML | Terminated | NCT02177812 |
| LSD1 inhibitor | INCB059872 + ATRA or azacitidine | 1 | R/R AML, newly diagnosed AML | Recruiting | NCT02712905 |
| LSD1 inhibitor | IMG-7289 ± ATRA | 1 | AML and MDS | Completed | NCT02842827 |
| LSD1 inhibitor | Tranylcypromine + ATRA and cytarabine | 1 | AML and MDS | Recruiting | NCT02717884 |
| LSD1 inhibitor | Tranylcypromine + ATRA | 1 | AML and MDS | Active, not recruiting | NCT02273102 |
| PRMT5 inhibitor | GSK3326595 | 1 | AML, MDS, CMML | Recruiting | NCT03614728 |
| Class . | Agent . | Phase . | Target population . | Status . | Identifier . |
|---|---|---|---|---|---|
| HMA | Guadecitabine | 3 | R/R AML | Active, not recruiting | NCT02920008 |
| HMA + DLI | Guadecitabine + DLI | 2 | AML or MDS relapsed post-AlloSCT | Recruiting | NCT02684162 |
| HMA | Guadecitabine | 3 | Newly diagnosed AML ineligible for intensive chemotherapy | Completed | NCT02348489 |
| HMA ± chemotherapy | Guadecitabine ± idarubicin | 2 | AML age ≥70 y | Active, not recruiting | NCT02096055 |
| HMA | Guadecitabine | 2 | AML with 20%-30% blasts or MDS refractory to azacitidine | Completed | NCT02197676 |
| HMA | Guadecitabine | 1/2 | AML, MDS | Completed | NCT01261312 |
| HMA + anti–PD-1 | Guadecitabine + atezolizumab | 1 | AML | Suspended | NCT02892318 |
| HDACi | Entinostat | 2 | AML age ≥60 y | Recruiting | NCT01305499 |
| HDACi + HMA | Entinostat + azacitidine | 1 | AML, MDS, CML | Active, not recruiting | NCT00101179 |
| HDACi + chemotherapy | Panobinostat + cytarabine and daunorubicin | 1 | AML, MDS age ≥60 | Active, not recruiting | NCT01463046 |
| HDACi + chemotherapy | Panobinostat + cytarabine and idarubicin | 1/2 | AML age ≥65 y | Completed | NCT00840346 |
| HDACi + chemotherapy | Panobinostat + cytarabine and idarubicin | 1 | AML age <65 y | Completed | NCT01242774 |
| HDACi + HMA | Panobinostat + azacitidine | 1 | AML <30% blasts, MDL, CMML, | Active, not recruiting | NCT00946647 |
| HDACi + HMA | Panobinostat + decitabine | 1/2 | AML age ≥60, MDS | Completed | NCT00691938 |
| HDACi + HMA | AR-42 + decitabine | 1 | AML | Completed | NCT01798901 |
| HDACi | Belinostat | 2 | AML age ≥60 | Completed | NCT00357032 |
| HDACi + HMA | Mocetinostat + azacitidine | 1/2 | AML, MDS | Completed | NCT00324220 |
| HDACi | Romidepsin | 2 | R/R AML | Completed | NCT00062075 |
| mIDH1 inhibitor | BAY1436032 | 1 | mIDH1 AML | Completed | NCT03127735 |
| mIDH1 inhibitor | Ivosidenib + venetoclax | 1/2 | mIDH1 AML, MDS/MPN | Recruiting | NCT03471260 |
| mIDH1 inhibitor ± HMA, chemotherapy | FT-2102 ± azacitidine or cytarabine | 1/2 | mIDH1 AML, MDS | Recruiting | NCT02719574 |
| mIDH2 inhibitor + HMA | Enasidenib + azacitidine | 2 | R/R mIDH2 AML | Recruiting | NCT03683433 |
| mIDH1, mIDH2 inhibitors + chemotherapy | Ivosidenib or enasidenib + cytarabine and daunorubicin or idarubicin | 1 | Newly diagnosed mIDH1 or mIDH2 AML | Active, not recruiting | NCT02632708 |
| mIDH1, mIDH2 inhibitors + HMA | Ivosidenib or enasidenib + azacitidine | 1/2 | Newly diagnosed mIDH1 or mIDH2 AML ineligible for intensive chemotherapy | Active, not recruiting | NCT02677922 |
| mIDH1 inhibitor + HMA | Ivosidenib or placebo + azacitidine | 3 | Newly diagnosed mIDH1 AML | Recruiting | NCT03173248 |
| BETi | OTX015 (MK-8628) | 1 | AML, ALL, NHL, MM | Completed | NCT01713582 |
| BETi ± HMA | FT-1101 ± azacitidine | 1 | R/R AML | Recruiting | NCT02543879 |
| BETi | GSK525762 | 1 | AML, NHL, MM | Recruiting | NCT01943851 |
| BETi | INCB054329 | 1/2 | AML, MDS/MPN, MM, solid tumor, or lymphoma | Completed | NCT02431260 |
| BETi ± BCL2 inhibitor | ABBV-075 +/− venetoclax | 1 | AML, MM, solid tumors, NHL | Active, not recruiting | NCT02391480 |
| EZH 1/2 inhibitor | DS-3201b | 1 | AML or ALL | Recruiting | NCT03110354 |
| BETi | RO6870810 | 1 | R/R AML and MDS | Completed | NCT02308761 |
| BETi | OTX015 + azacitidine | 1b/2 | Newly diagnosed AML ineligible for intensive chemotherapy | Withdrawn | NCT02303782 |
| LSD1 inhibitor | GSK2879552 | 1 | R/R AML | Terminated | NCT02177812 |
| LSD1 inhibitor | INCB059872 + ATRA or azacitidine | 1 | R/R AML, newly diagnosed AML | Recruiting | NCT02712905 |
| LSD1 inhibitor | IMG-7289 ± ATRA | 1 | AML and MDS | Completed | NCT02842827 |
| LSD1 inhibitor | Tranylcypromine + ATRA and cytarabine | 1 | AML and MDS | Recruiting | NCT02717884 |
| LSD1 inhibitor | Tranylcypromine + ATRA | 1 | AML and MDS | Active, not recruiting | NCT02273102 |
| PRMT5 inhibitor | GSK3326595 | 1 | AML, MDS, CMML | Recruiting | NCT03614728 |
ALL, acute lymphoblastic leukemia; AlloSCT, allogeneic stem cell transplantation; ATRA, all-trans retinoic acid; DLI, donor leukocyte infusion; MM, multiple myeloma; MPN, myeloproliferative neoplasm; NHL, non-Hodgkin lymphoma; PRMT5, protein arginine methyltransferase 5; ±, with or without.