Clinical Trials Search
A Phase III Multicenter, Randomized, Open Label Study of APR-246 in Combination with Azacitidine Versus Azacitidine Alone for the Treatment of TP53 Mutant Myelodysplastic Syndromes
A Phase III, multicenter, randomized study to compare the rate of complete response (CR) and duration of CR, in patients with TP53-mutated MDS who will receive APR-246 and azacitidine or azacitidine alone.
Primary Objective: 1. To compare the complete response rate, defined as the proportion of patients who achieve complete remission (CR), and duration of CR with APR-246 + azacitidine treatment vs. azacitidine only. Secondary Objectives: To measure the following with APR-246 + azacitidine treatment vs. azacitidine alone: 1. Overall response rate (ORR): CR, PR, mCR, mCR+HI, HI 2. Duration of Response (DOR) 3. Rate and time to acute myeloid leukemia (AML) transformation 4. Overall survival (OS) 5. Progression free survival (PFS) 6. Transition rate to hematopoietic stem cell transplant (HSCT) 7. Safety profile 8. Rate of red blood cell (RBC) and/or platelet transfusion independence (TI) for 56 days (8 weeks) 9. Pharmacokinetics of APR-246 and influence of APR-246 on azacitidine pharmacokinetics