Major monotherapy trials of epigenetic-directed therapies
| Class . | Agent . | Trial phase . | Target population . | Patients, n . | Response . | Identifier . |
|---|---|---|---|---|---|---|
| HMA + BCL2 inhibitor | Azacitidine + venetoclax | 1 | Elderly, treatment-naive AML | 145 | 67% CR/CRi | NCT02203773 |
| HMA + anti–PD-1 | Azacitidine + nivolumab | 1b/2 | R/R AML | 51 | 18% CR/CRi | NCT02397720 |
| HMA + chemotherapy | Azacitidine + chemotherapy vs chemotherapy alone | 3 | Newly diagnosed AML | 209 | 48% and 52% CR, respectively | NCT00915252 |
| HMA + mIDH inhibitor | Azacitidine + enasidenib or ivosidenib | 1b/2* | IDH-mutated newly diagnosed AML ineligible for intensive chemotherapy | 13 | 33% CR enasidenib, 43% CR ivosidenib | NCT02677922 |
| HDACi + chemotherapy | Vorinostat + idarubicin and cytarabine | 2 | Newly diagnosed AML or higher-risk MDS | 75 | 76% CR and 9% CRp | NCT00656617 |
| HMA + HDACi | Vorinostat + azacitidine | 1 | AML or MDS | 6 AML, 14 MDS | 45% CR and 9% CRi | NCT00392353 |
| HMA + HDACi | Vorinostat + azacitidine vs azacitidine alone | 2 | AML and high-risk MDS ineligible for intensive chemotherapy | 217 AML, 42 MDS | 26% and 22% CR/CRi/mCR, respectively (P = .49)† | NCT01617226 |
| HMA + HDACi | Azacitidine + pracinostat | 2 | Newly diagnosed AML ineligible for intensive chemotherapy | 50 | 42.0% CR | NCT01912274 |
| HMA + HDACi | Romidepsin + azacitidine | 1 | Newly diagnosed or R/R AML ineligible for intensive chemotherapy | 46 | 38.9% CR/CRi | ISRCTN69211255 |
| mIDH inhibitor + chemotherapy | Enasidenib or ivosidenib + chemotherapy | 1 | Newly diagnosed IDH1- or IDH2-mutated AML | 65 | 69.6% CR/CRi/CRp (ivosidenib) and 62.2% CR/CRi/CRp (enasidenib) | NC02632708 |
| Class . | Agent . | Trial phase . | Target population . | Patients, n . | Response . | Identifier . |
|---|---|---|---|---|---|---|
| HMA + BCL2 inhibitor | Azacitidine + venetoclax | 1 | Elderly, treatment-naive AML | 145 | 67% CR/CRi | NCT02203773 |
| HMA + anti–PD-1 | Azacitidine + nivolumab | 1b/2 | R/R AML | 51 | 18% CR/CRi | NCT02397720 |
| HMA + chemotherapy | Azacitidine + chemotherapy vs chemotherapy alone | 3 | Newly diagnosed AML | 209 | 48% and 52% CR, respectively | NCT00915252 |
| HMA + mIDH inhibitor | Azacitidine + enasidenib or ivosidenib | 1b/2* | IDH-mutated newly diagnosed AML ineligible for intensive chemotherapy | 13 | 33% CR enasidenib, 43% CR ivosidenib | NCT02677922 |
| HDACi + chemotherapy | Vorinostat + idarubicin and cytarabine | 2 | Newly diagnosed AML or higher-risk MDS | 75 | 76% CR and 9% CRp | NCT00656617 |
| HMA + HDACi | Vorinostat + azacitidine | 1 | AML or MDS | 6 AML, 14 MDS | 45% CR and 9% CRi | NCT00392353 |
| HMA + HDACi | Vorinostat + azacitidine vs azacitidine alone | 2 | AML and high-risk MDS ineligible for intensive chemotherapy | 217 AML, 42 MDS | 26% and 22% CR/CRi/mCR, respectively (P = .49)† | NCT01617226 |
| HMA + HDACi | Azacitidine + pracinostat | 2 | Newly diagnosed AML ineligible for intensive chemotherapy | 50 | 42.0% CR | NCT01912274 |
| HMA + HDACi | Romidepsin + azacitidine | 1 | Newly diagnosed or R/R AML ineligible for intensive chemotherapy | 46 | 38.9% CR/CRi | ISRCTN69211255 |
| mIDH inhibitor + chemotherapy | Enasidenib or ivosidenib + chemotherapy | 1 | Newly diagnosed IDH1- or IDH2-mutated AML | 65 | 69.6% CR/CRi/CRp (ivosidenib) and 62.2% CR/CRi/CRp (enasidenib) | NC02632708 |