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Trametinib in Treating Patients with Relapsed or Refractory Juvenile Myelomonocytic Leukemia

Active: Yes
Cancer Type: Cancer-Related Syndrome
Hematopoietic Malignancies
Unknown Primary
NCT ID: NCT03190915
Trial Phases: Phase II Protocol IDs: ADVL1521 (primary)
Eligibility: 1 Months - 21 Years, Male and Female Study Type: Treatment
Study Sponsor: Children's Oncology Group
NCI Full Details:


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. 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.

Treatment Sites in Georgia

Aflac Cancer and Blood Disorders Center of Children’s at Egleston
1405 Clifton Road NE
3rd Floor
Atlanta, GA 30322

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