A Phase 2 Study of the MEK Inhibitor Trametinib (NSC# 763093) in Children With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

    Not Recruiting
  • End date
    Mar 31, 2027
  • participants needed
  • sponsor
    National Cancer Institute (NCI)
Updated on 15 September 2023
ct scan
white blood cell count
absolute neutrophil count
ejection fraction
monoclonal antibodies
biologic agent
measurable disease
total serum bilirubin
growth factor
transplant conditioning
hematopoietic growth factors
tumor cells
chromosomal abnormality


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 refractory JMML.

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 refractory JMML.


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.

Condition Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Treatment laboratory biomarker analysis, computed tomography, magnetic resonance imaging, pharmacological study, Trametinib, Bone Marrow Aspiration and Biopsy
Clinical Study IdentifierNCT03190915
SponsorNational Cancer Institute (NCI)
Last Modified on15 September 2023

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