PLX51107 and Azacitidine in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Summary

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Description

PRIMARY OBJECTIVE: I. To determine the minimum safe and biologically-effective dose of the combination of PLX51107 and azacitidine (AZA). SECONDARY OBJECTIVES: I. To determine overall response rate (ORR) rate including CR (complete remission) + CRp (complete remission with incomplete platelet recove...

PRIMARY OBJECTIVE: I. To determine the minimum safe and biologically-effective dose of the combination of PLX51107 and azacitidine (AZA). SECONDARY OBJECTIVES: I. To determine overall response rate (ORR) rate including CR (complete remission) + CRp (complete remission with incomplete platelet recovery) + CRi (complete remission with incomplete count recovery) + CRh (complete remission with partial hematologic recovery), partial remission (PR) within 3 months of treatment initiation of PLX51107 and AZA combination in patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). II. To investigate correlations of response to this combination with a pre-therapy, on-therapy, and progression 81-gene panel of gene mutations in AML. III. To determine the duration of response (DOR), event-free survival (EFS), overall survival (OS), and number of patients bridged to stem cell transplant (SCT) and median duration to SCT from the initiation of the combination. OUTLINE: This is a dose-escalation study of PLX51107. Patients receive PLX51107 orally (PO) once daily (QD) on days 1-21 and azacitidine subcutaneously (SC) or intravenously (IV) over 15 minutes on days 8-14 and 22-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days, then every 6-12 months for 5 years.

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