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

  • STATUS
    Not Recruiting
  • End date
    Mar 31, 2027
  • participants needed
    24
  • sponsor
    National Cancer Institute (NCI)
Updated on 15 September 2023
ct scan
fludarabine
hydroxyurea
cytarabine
white blood cell count
bcr/abl
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

Summary

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.

Description

PRIMARY OBJECTIVE:

I. To determine the objective response rate to trametinib in children with recurrent or refractory juvenile myelomonocytic leukemia (JMML).

SECONDARY OBJECTIVES:

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.

EXPLORATORY OBJECTIVE:

I. To describe the distribution of JMML diagnostic criteria in children with recurrent or refractory JMML.

OUTLINE

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.

Details
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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