Last updated on January 2020

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia


Brief description of study

This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back or does not respond to treatment. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed Study 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. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up annually for up to 5 years.

Clinical Study Identifier: NCT03190915

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Mount Sinai Hospital

New York, NY United States
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Children's Hospital of Alabama

Birmingham, AL United States
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Nemours Children's Hospital

Orlando, FL United States
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C S Mott Children's Hospital

Ann Arbor, MI United States
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Seattle Children's Hospital

Seattle, WA United States
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Arkansas Children's Hospital

Little Rock, AR United States
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Kaiser Permanente-Oakland

Oakland, CA United States
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