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A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Study of Trametinib and Azacitidine for new juvenile leukemia cases.

Recruiting
1-21 years
All
Phase 1/2

This study is for children and young adults (1 month to 21 years old) with a type of blood cancer called Juvenile Myelomonocytic Leukemia (JMML). Researchers are testing if two drugs, trametinib and azacitidine, are safe and work well together.

JMML is a rare leukemia, which means a type of cancer of the blood. It can be either lower-risk or higher-risk. In this study, lower-risk patients will take trametinib and azacitidine every day for 28 days, and this can happen up to 12 times. Higher-risk patients will also take two more drugs, fludarabine and cytarabine, for up to 2 rounds.

  • The study could last up to a year for lower-risk patients and a few months for higher-risk ones.
  • Participants must have certain blood counts and genetic indicators of JMML.
  • Participants can't have had certain other treatments before joining.

Parents or guardians should consider the length of the study and if their child has any conditions that might exclude them before deciding to participate.

Study details
    Leukemia
    Juvenile Myelomonocytic
    JMML
    JCML
    Neurofibromatosis 1
    CBL Syndrome

NCT05849662

Therapeutic Advances in Childhood Leukemia Consortium

25 April 2026

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