This phase II trial studies how well trametinib works in treating patients with juvenile
myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment
(refractory). Trametinib may stop the growth of tumor cells by blocking some of the enzymes
needed for cell growth.
I. To determine the objective response rate to trametinib in children with recurrent or
refractory juvenile myelomonocytic leukemia (JMML).
I. To further define and describe the toxicities of single agent trametinib in children with
recurrent or refractory JMML.
II. To further characterize the pharmacokinetics of trametinib in children with recurrent or
III. To prospectively evaluate mutant allele burden as a marker of disease activity in JMML.
IV. To measure the rate of complete responses in children with recurrent or refractory JMML.
V. To measure the duration of response among responders.
I. To describe the distribution of JMML diagnostic criteria in children with recurrent or
Patients receive trametinib orally (PO) once daily (QD) on days 1-28 of each cycle. Treatment
repeats every 28 days for up to 12 cycles in the absence of disease progression or
unacceptable toxicity. Patients undergo computed tomography (CT) or magnetic resonance
imaging (MRI) at baseline and may undergo a bone marrow aspiration or biopsy at baseline, on
day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.
After completion of study treatment, patients are followed up annually for up to 5 years.
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
laboratory biomarker analysis,
magnetic resonance imaging,
Bone Marrow Aspiration and Biopsy
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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