Phase 1 Dose Escalation Study of CPX-351 for Patients With Int-2 or High Risk IPSS Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia After Failure to Hypomethylating Agents
This phase I trial studies best dose and side effects of liposome-encapsulated daunorubicin-cytarabine (CPX-351) and how well it works in treating patients with high risk myelodysplastic syndrome or chronic myelomonocytic leukemia that has come back or has not responded to treatment. Drugs used in chemotherapy, such as liposome-encapsulated daunorubicin-cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
• Diagnosis of MDS or chronic myelomonocytic leukemia (CMML) according to World Health Organization (WHO)
• Patients are either not eligible for or choose not to proceed with a stem cell transplant at the time of enrollment
• MDS and CMML classified by International Prognostic Scoring System (IPSS) as intermediate-2/high risk with excess blasts \> 5%, or with 10-19% bone marrow blasts
• No response following at least 4 cycles of therapy or relapse after initial CR, partial response (PR), or HI or progression after any number of cycles of either azacitidine, decitabine, guadecitabine or ASTX727 (oral decitabine) as single agents or in combination with other investigational agents
• Patient (or patient's legally authorized representative) must have signed an informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study
• Total bilirubin \< 3 mg/dL (will allow less than 5 x upper limit of normal \[ULN\] if Gilbert's at investigator's discretion)
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =\< 3 x ULN
• Serum creatinine clearance \> 30 mL/min and no end/stage renal disease
• Hydroxyurea for control of leukocytosis or use of hematopoietic growth factors (eg, granulocyte-colony stimulating factor \[G-CSF\], granulocyte-macrophage colony-stimulating factor \[GM-CSF\], procrit, aranesp, thrombopoietins) is allowed at any time prior to or during study if considered to be in the best interest of the patient