Acute Myeloid Leukemia Treatment with CPX-351 and Gilteritinib – Phase III

AAML1831: A Study to Compare Standard Chemotherapy to Therapy with CPX-351 and/or Gilteritinib for Patients with Newly Diagnosed AML with or without FLT3 Mutations

Protocol Description

Through this multi-center international study organized by the Children’s Oncology Group, researchers will compare standard chemotherapy for acute myeloid leukemia (AML) to treatment in which the investigational drug CPX-351 is used. For patients with the FLT3 gene mutation, the drug gilteritinib also will be tested. AML is a cancer of the bone marrow. CPX-351, which is approved for adult use, has been shown to stay in bone marrow longer and poses less of a risk for heart problems than traditional chemotherapy drugs. Gilteritinib, which is also approved for treatment of adults, has been shown to block the effects of an abnormal FLT3 gene in older AML patients, but has not yet been proven in children.

Study Level: III

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling patients through age 21 who have been diagnosed with AML.
Males and Females: Up through 21 years of age

Requirements

Through random selection, some participants will receive standard chemotherapy and others will receive the drug CPX-351 in place of certain traditional chemotherapy drugs during the first phase of treatment, which is called induction. Gilteritinib will be introduced for applicable participants during induction and continued for the second phase of chemotherapy, called intensification, which does not involve GPX-351. Depending on risk stratification, the intensification phase may have multiple stages or may include stem cell transplantation for certain participants.
Visits: The number varies based on participant response to therapy.
Duration: About 6 months in most cases.

Status: Open to Enrollment

Source(s) of Support

Children’s Oncology Group (AAML1831)
National Cancer Institute

Primary Investigator

Jean M. Tersak, MD

Contact Information

For more information about the study or enrollment, please contact the UPMC Children’s Hospital of Pittsburgh Hematology-Oncology research team, at:
HOCHPPhaseI-II@upmc.edu.