This study is being done to evaluate the safety and effectiveness of APTO-253 for the
treatment of patients with the condition of acute myelogenous leukemia (AML) or
myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no
longer effective, or can no longer be administered safely or poses a risk for your general
well being.
This is a multicenter, open-label, Phase Ia/b dose escalation study of safety,
pharmacodynamics, and pharmacokinetics of APTO-253 in ascending cohorts (3+3 design) to
determine the MTD or recommended dose in patients with relapsed or refractory acute
myelogenous leukemia (AML) or high-risk MDS patients. This is to be followed by a cohort
expansion phase at the MTD or recommended dose.
Inclusion Criteria:
- Patients ≥18 years old
- Life expectancy of at least 2 months
- Off previous cancer therapy for at least 14 days, or 5 half-lives for noncytotoxic
agents prior to first study treatment administration
- Patients must have a calculated creatinine clearance >60 mL/min
- Acceptable hematologic, renal and liver functions and coagulation status parameters
Exclusion Criteria:
- Patients with GVHD requiring systemic immunosuppressive therapy
- Uncontrolled leptomeningeal disease, auto-immune hemolytic anemia and uncontrolled and
clinical significant disease related metabolic disorder
- Clinically significant intravascular coagulation
- Treatment with other investigational drugs within 14 days prior to first study
treatment administration