Juvenile Myelomonocytic Leukemia Treatment using Trametinib – Phase II

ADVL1521: Study of the MEK Inhibitor Trametinib in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia

Protocol Description

This multi-center Children’s Oncology Group initiative, studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia (JMML) that has come back or does not respond to treatment. There is no standard treatment for this condition, however researchers believe trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Trametinib is approved by the Food and Drug Administration (FDA) for the treatment of adults with certain types of cancers.

Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling patients who have been diagnosed with JMML.
Males and Females: 1 month to 21 years


In 28-day cycles, study participants will take the study medication orally once daily. Participants will have evaluations, including blood sampling, at the end of the first cycle and every second cycle thereafter. Participants may remain in the study for up to 12 cycles, barring serious side effects or worsening of the condition.
Visits: 14 (Every 2 weeks in months 1 and 2, followed by monthly visits)
Duration: Up to 1 year

Status: Open to Enrollment

Source(s) of Support

Children’s Oncology Group (ADVL1521)

Primary Investigator

Jean Tersak, MD

Contact Information

For more information about the study or enrollment, please contact:
Sharon Dibridge, Clinical Research Coordinator