Treatment protocols in acute myeloid leukemia (AML) have been associated with high rates of acute treatment-associated morbidity and mortality. Phase I trials of novel liposomal nanoparticle-based targeted delivery systems for chemotherapy (NANOLEUK) have shown safety in AML patients. We aim to determine that NANOLEUK improves survival and decreases adverse events as compared with the standard chemotherapy in AML patients.

We will conduct an international, multicenter, randomized controlled, double-blind, 24 month-long trial involving 224 patients recruited from 10 hematology-oncology centers in four countries (Brazil, Peru, Colombia and United States) diagnosed with de novo non-promyelocytic AML. The patients will be randomly assigned in a 1:1 ratio toreceive 1) NANOLEUK (liposomal cytarabine and daunorubicin) or 2) standard chemotherapy. The study will use a central computer assisted block randomization scheme and an interactive web response system. The primary outcome will be median survival time at 12 months of follow-up. Secondary outcomes will include complete remission, adverse events, quality of life and hospitalization length.

We anticipate that the results will show improved median survival time at 12 months of follow-up in the NANOLEUK group, with a reduction in treatment-associated adverse events and improvement in quality of life. This is the first randomized controlled trial to establish the efficacy of NANOLEUK compared with standard chemotherapy in AML patients. The efficacy and safety profile of NANOLEUK will have a broad clinical implication in AML treatment that should be confirmed in a follow-up phase III trial.