A Phase II Study of CPX-351 for Treatment of AML or Higher Risk MDS Relapsed or Refractory to Prior Therapy With Hypomethylating (HMA) Agent
This phase II clinical trial studies how well liposomal cytarabine-daunorubicin CPX-351 (CPX-351) works in treating patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome. Drugs used in chemotherapy, such as liposomal cytarabine-daunorubicin CPX-35, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- drug : liposomal cytarabine-daunorubicin CPX-351
- other : laboratory biomarker analysis
Phase: Phase 2
Ages Eligible For Study:
- Ability to understand and voluntarily give informed consent - Age ? 60 - Pathological diagnosis of AML (by WHO criteria) or higher risk MDS (includes int-2 and high risk MDS by IPSS) along with one of the following: - Patients with de novo or secondary MDS with progression/refractoriness after HMA treatment who have not transformed to AML - Patients with MDS and prior HMA treatment for MDS who transform to AML - Patients with AML who are refractory/relapsed after HMA therapy for their AML are eligible - Life expectancy > 1 month - Eastern Cooperative Oncology Group (ECOG) performance status 0-2 - Able to adhere to the study visit schedule and other protocol requirements - Laboratory values fulfilling the following: - Serum creatinine < 2.0 mg/dL - Serum total bilirubin ? 2.5 mg/dL. Note, patients with Gilbert's syndrome may have elevated bilirubin at baseline prior to diagnosis with AML or MDS. Patients with Gilbert's syndrome are included if their total bilirubin is ? 2 times their baseline total bilirubin. - Serum alanine aminotransferase or aspartate aminotransferase < 3 times ULN - Cardiac ejection fraction ? 45% by echocardiography (transthoracic echocardiography) or MUGA scan - Patients with second malignancies may be eligible at discretion of PI given acute life threatening nature of untreated AML or higher risk MDS. Patients maintained on long-term non-chemotherapy treatment, e.g., hormonal therapy, are also eligible.