Trametinib for Juvenile Myelomonocytic Leukemia
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications before participating. You cannot be on other anti-cancer agents, investigational drugs, or medications for preventing organ rejection. If you are on corticosteroids, you must be on a stable or decreasing dose for at least 7 days before enrolling. Hydroxyurea can be continued until 24 hours before starting the trial treatment.
How is the drug trametinib unique in treating juvenile myelomonocytic leukemia?
What data supports the effectiveness of the drug Trametinib for treating Juvenile Myelomonocytic Leukemia?
Research shows that combining Trametinib, a MEK inhibitor, with other treatments like 5-azacitidine can help reduce leukemia-related issues in a mouse model of Juvenile Myelomonocytic Leukemia. Additionally, two patients with specific genetic mutations in JMML showed positive responses when treated with Trametinib, 5-azacitidine, and chemotherapy.13456
Who Is on the Research Team?
Elliot Stieglitz
Principal Investigator
Children's Oncology Group
Are You a Good Fit for This Trial?
This trial is for young patients under 22 years with juvenile myelomonocytic leukemia (JMML) that's relapsed or not responding to treatment. They must have recovered from previous treatments, be able to swallow pills, and meet specific health criteria like heart function and normal blood levels.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive trametinib orally once daily on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles.
Follow-up
Participants are monitored for safety and effectiveness after treatment completion
What Are the Treatments Tested in This Trial?
Interventions
- Trametinib
Trametinib is already approved in European Union, United States, Canada, Japan for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Cancer Institute (NCI)
Lead Sponsor
Children's Oncology Group
Collaborator