Outcomes of phase 2 and 3 trials of oral decitabine/cedazuridine
| Characteristic . | Phase 2 (NCT02103478) ASTX727-01-B45 . | Phase 3 (NCT03306264) ASTX727-0246,47 . |
|---|---|---|
| Total patients, N | 80 | 133 |
| Mean age (range), y | 71 (32-90) | 71 (44-88) |
| MDS (intermediate-1), n (%) | 35 (44) | 11 (8) |
| MDS (intermediate-2), n (%) | 19 (24) | 85 (64) |
| MDS (high risk), n (%) | 9 (11) | 21 (16) |
| CMML, n (%) | 17 (21) | 16 (12) |
| Median number of cycles (range) | 7 (1-29) | 8 (1-18) |
| Oral/IV ratio of geometric LSM 5-d AUC, % | 97.6 | 98.9 |
| Difference (oral-IV) in mean maximum LINE-1 demethylation, % | 0.017-1.079 | 0.7-0.8 |
| Patients with CR, n (%) | 17 (21) | 29 (22) |
| Patients with PR, n (%) | 0 | 0 |
| Patients with mCR, n (%) | 18 (22) | 43 (32) |
| Overall response (CR + PR + mCR + HI), n (%) | 48 (60) | 82 (62) |
| Median follow-up, mo | 24 | 24.7 |
| Median overall survival, mo | 18.3 | NR |
| Most common grade ≥3 TEAEs, % | Neutropenia: 46 | Neutropenia: 52 |
| Thrombocytopenia: 38 | Thrombocytopenia: 50 | |
| Febrile neutropenia: 29 | Anemia: 40 | |
| Leukopenia: 24 | Febrile neutropenia: 26 | |
| Anemia: 22 | Leukopenia: 21 | |
| Pneumonia: 13 | Pneumonia: 12 | |
| Sepsis 10 |
| Characteristic . | Phase 2 (NCT02103478) ASTX727-01-B45 . | Phase 3 (NCT03306264) ASTX727-0246,47 . |
|---|---|---|
| Total patients, N | 80 | 133 |
| Mean age (range), y | 71 (32-90) | 71 (44-88) |
| MDS (intermediate-1), n (%) | 35 (44) | 11 (8) |
| MDS (intermediate-2), n (%) | 19 (24) | 85 (64) |
| MDS (high risk), n (%) | 9 (11) | 21 (16) |
| CMML, n (%) | 17 (21) | 16 (12) |
| Median number of cycles (range) | 7 (1-29) | 8 (1-18) |
| Oral/IV ratio of geometric LSM 5-d AUC, % | 97.6 | 98.9 |
| Difference (oral-IV) in mean maximum LINE-1 demethylation, % | 0.017-1.079 | 0.7-0.8 |
| Patients with CR, n (%) | 17 (21) | 29 (22) |
| Patients with PR, n (%) | 0 | 0 |
| Patients with mCR, n (%) | 18 (22) | 43 (32) |
| Overall response (CR + PR + mCR + HI), n (%) | 48 (60) | 82 (62) |
| Median follow-up, mo | 24 | 24.7 |
| Median overall survival, mo | 18.3 | NR |
| Most common grade ≥3 TEAEs, % | Neutropenia: 46 | Neutropenia: 52 |
| Thrombocytopenia: 38 | Thrombocytopenia: 50 | |
| Febrile neutropenia: 29 | Anemia: 40 | |
| Leukopenia: 24 | Febrile neutropenia: 26 | |
| Anemia: 22 | Leukopenia: 21 | |
| Pneumonia: 13 | Pneumonia: 12 | |
| Sepsis 10 |
TEAEs, treatment-emergent adverse events.