This phase I trial tests the safety, side effects, and best dose of imetelstat in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) that has not responded to previous treatment (refractory) or that has come back after a period of improvement (recurrent). Imetelstat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as fludarabine and 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. Giving imetelstat in combination with fludarabine and cytarabine may work better in treating patients with refractory or recurrent AML, MDS, and JMML.
Eligibility Criteria
Between 12 months and 18 years old
Relapsed or refractory AML, MDS, or JMML
Have not received imetelstat
Major Inclusion Criteria
Eligible patients must be between the ages of 1 to 18 years at the time of study enrollment with Lansky/Karnofsky performance status of ≥50.
2nd or greater relapse of AML, 1st or greater relapse of MDS/JMML
Refractory disease is allowed
Down Syndrome is allowed
Therapy related AML is allowed
Standard organ function criteria
Patients with leukemia must have platelet count >25,000/µL and hemoglobin >8 g/dL (transfusions allowed)
