Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03745716 | APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS) | PHASE3 | COMPLETED | 154 | — | — | Jan 11, 2019 | Jan 14, 2022 | Mar 18, 2025 | 27 | United States, France |
To compare the complete response rate, defined as the proportion of patients who achieve complete remission (CR) with APR 246 + azacitidine treatment vs. azacitidine only.
| Arm | Type | Description |
|---|---|---|
| Experimental arm: APR-246 + azacitidine | EXPERIMENTAL | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): |
| Control arm: Azacitidine | EXPERIMENTAL | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): |
| Name | Type | Description |
|---|---|---|
| APR-246 + azacitidine | DRUG | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): Experimental arm: APR-246 + azacitidine; or Control arm: Azacitidine |
| Azacitidine | DRUG | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): Experimental arm: APR-246 + azacitidine; or Control arm: Azacitidine |
Inclusion Criteria: * Signed Informed Consent (ICF) and is able to comply with protocol requirements * Documented diagnosis of MDS, according to World Health Organization (WHO) classification * Patient has adequate organ function as defined by the following laboratory values: 1. Creatinine clear...