ACCL0933, A Randomized Open-Label Trial of Caspofungin versus Fluconazole to Prevent Invasive Fungal Infections in Children Undergoing Chemotherapy for Acute Myeloid Leukemia (AML)
Invasive fungal infections (IFI) have steadily increased over the past 2 decades in children with acute myeloid leukemia (AML). Over 50% of infectious-related deaths in this population are attributable to IFI. These dismal numbers are despite empiric therapy and treatment with the best available agents. The failure of these strategies to impact morbidity and mortality from IFI makes a strong case for the exploration of preventive strategies. This study will utilize a 2-arm, open-label randomized design to evaluate the efficacy of prophylaxis with caspofungin in comparison with fluconazole in children with de novo, relapsed or secondary AML. Study agent will begin following completion of each course of chemotherapy and continue through periods of neutropenia. This study also seeks to evaluate the usefulness of the Platelia EIA Aspergillus galactomannan (GM) assay as well as beta-D glucan testing in early diagnosis of IFI. ACCL0933 will also explore the relationship between proven or probable IFI and single nucleotide polymorphisms (SNP) of genes involved in immunity and develop predictive models of IFI that will be useful in the early identification of susceptible patients.
1) Patients must be >= 3 months and <= 30 years at the time of enrollment.
2) Patients must have one of the following diagnoses and/or treatment plans:
- Newly diagnosed de novo AML
- First or subsequent relapse of AML
- Secondary AML
- Treatment with institutional standard AML therapy in those without AML (for example myelodysplastic syndrome, bone marrow blasts > 5% or
3) Adequate renal function
4) Adequate liver function
5) All patients and/or their parents or legal guardians must sign a written informed consent.
6) All institutional, FDA, and NCI requirements for human studies must be met.