A Phase 1-2 Study of Trametinib and Azacitidine With or Without Chemotherapy for Young People With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Full Title

Risk Stratified Treatment for Patients with Newly Diagnosed Juvenile Myelomonocytic Leukemia: A Phase I/II Non-randomized Study of Trametinib and Azacitidine with or without Chemotherapy (IND # 164058) (TACL_T2020-004) (CHLA IRB)

Purpose

Researchers are assessing trametinib and azacitidine alone and with other drugs to treat leukemia in young people. The people in this study have newly diagnosed juvenile myelomonocytic leukemia (JMML).

Children with JMML that has a low chance of coming back after treatment will get trametinib and azacitidine. Those with high-risk JMML will get trametinib, azacitidine, fludarabine, and cytarabine. The intensity of treatment will vary depending on the DNA changes in each patient’s cancer.

Who Can Join

To join this study, there are a few conditions. Your child must:

  • Be newly diagnosed with JMML.
  • Be age 1 month to 21 years.

Contact

For more information or to see if your child can join this study, please call 833-MSK-KIDS.

Protocol

25-137

Phase

Phase I/II (phases 1 and 2 combined)

Disease Status

Newly Diagnosed

Investigator

Co-Investigators

ClinicalTrials.gov ID

NCT05849662