Single-arm and randomized trial response rates and expected response rates
| Combination . | NCT . | Single-arm response . | Randomized endpoint . | Randomized response . | Expected response (phase 1/2) . |
|---|---|---|---|---|---|
| Azacitidine + entinostat | Phase 1: NCT00101179 Phase 2: NCT00313586 | 3/30 CR 4/30 PR 7/30 HI | TN response rate: 30% vs 16% historical | Aza: 9/74 CR 24/74 TN Aza + entinostat: 8/75 CR 20/75 TN | Unlikely to improve on CR Unlikely to improve on HI |
| Decitabine + valproic acid | Phase 1/2: NCT00075010 Phase 2 randomized: NCT00414310 | 10/53 CR | Response rate | Decitabine: 22/70 CR Decitabine + VPA: 29/79 CR | Unlikely to improve on CR |
| Azacitidine + lenalidomide | Phase 1: NCT00352001 Phase 2: NCT01522976 | 16/36 CR 20/36 HI | ORR including HI | Aza: 24/92 CR 14/92 HI Aza + lenalidomide: 24/93 CR 25/93 HI | Moderately improved CR (>30%, P = .047) Unlikely to have 50% CR |
| Azacitidine + vorinostat | Phase 1: NCT00392353 Phase 2: NCT01522976 | 10/33 CR | ORR including HI | Aza: 24/92 CR Aza + vorinostat: 17/92 CR | Unlikely to improve on CR |
| Azacitidine + eprenetapopt | 20/40 CR | CR rate | Aza: 17/76 22.4% Aza + eprenetapopt: 26/78, 33.3% | Moderately better CR (>30%, P = .006) Unlikely to have 50% CR | |
| Azacitidine + pevonedistat | Phase 2: NCT02610777 Phase 3: NCT03268954 | 22/55 CR EFS and OS NS by ITT | EFS primary endpoint | Did not meet EFS benefit | Unlikely to improve on CR |
| Combination . | NCT . | Single-arm response . | Randomized endpoint . | Randomized response . | Expected response (phase 1/2) . |
|---|---|---|---|---|---|
| Azacitidine + entinostat | Phase 1: NCT00101179 Phase 2: NCT00313586 | 3/30 CR 4/30 PR 7/30 HI | TN response rate: 30% vs 16% historical | Aza: 9/74 CR 24/74 TN Aza + entinostat: 8/75 CR 20/75 TN | Unlikely to improve on CR Unlikely to improve on HI |
| Decitabine + valproic acid | Phase 1/2: NCT00075010 Phase 2 randomized: NCT00414310 | 10/53 CR | Response rate | Decitabine: 22/70 CR Decitabine + VPA: 29/79 CR | Unlikely to improve on CR |
| Azacitidine + lenalidomide | Phase 1: NCT00352001 Phase 2: NCT01522976 | 16/36 CR 20/36 HI | ORR including HI | Aza: 24/92 CR 14/92 HI Aza + lenalidomide: 24/93 CR 25/93 HI | Moderately improved CR (>30%, P = .047) Unlikely to have 50% CR |
| Azacitidine + vorinostat | Phase 1: NCT00392353 Phase 2: NCT01522976 | 10/33 CR | ORR including HI | Aza: 24/92 CR Aza + vorinostat: 17/92 CR | Unlikely to improve on CR |
| Azacitidine + eprenetapopt | 20/40 CR | CR rate | Aza: 17/76 22.4% Aza + eprenetapopt: 26/78, 33.3% | Moderately better CR (>30%, P = .006) Unlikely to have 50% CR | |
| Azacitidine + pevonedistat | Phase 2: NCT02610777 Phase 3: NCT03268954 | 22/55 CR EFS and OS NS by ITT | EFS primary endpoint | Did not meet EFS benefit | Unlikely to improve on CR |
EFS, event-free survival; TN, trilineage; VPA, valproic acid.