10 Participants Needed

Trametinib for Juvenile Myelomonocytic Leukemia

Recruiting at 57 trial locations
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 4 JurisdictionsThis treatment is already approved in other countries

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?

Trametinib is unique because it targets the Ras/MAPK pathway, which is often hyperactivated in juvenile myelomonocytic leukemia due to mutations like PTPN11. This makes it different from traditional chemotherapy, which is not durably effective for this condition.12345

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?

ES

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

I am between 1 month and 22 years old.
My leukemia (JMML) has come back or is not responding to treatment.
I can do most activities but may need help.
See 2 more

Exclusion Criteria

Patients with a history of allergic reaction attributed to compounds of similar chemical or biologic composition to the MEK inhibitor, trametinib
I am taking medication to prevent organ rejection after a transplant.
Patients who are pregnant or breast-feeding
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive trametinib orally once daily on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles.

12 cycles (1 cycle = 28 days)
Bone marrow aspiration or biopsy at baseline, day 28 of cycles 1 and 2, all subsequent odd numbered cycles, and end of treatment

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

Annually for up to 5 years

What Are the Treatments Tested in This Trial?

Interventions

  • Trametinib
Trial Overview The study tests trametinib effectiveness in treating JMML that has returned or is refractory. Trametinib aims to halt tumor growth by inhibiting enzymes necessary for cell proliferation. The process includes bone marrow procedures alongside medication administration.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment (trametinib)Experimental Treatment2 Interventions

Trametinib is already approved in European Union, United States, Canada, Japan for the following indications:

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Approved in European Union as Mekinist for:
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Approved in United States as Mekinist for:
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Approved in Canada as Mekinist for:
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Approved in Japan as Mekinist for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Children's Oncology Group

Collaborator

Trials
467
Recruited
241,000+

Published Research Related to This Trial

Chronic myelomonocytic leukemia (CMML) shows increased sensitivity of myeloid progenitors to granulocyte macrophage-colony stimulating factor, indicating a need for targeted therapies.
Ruxolitinib, a JAK1 and JAK2 inhibitor, has been demonstrated to induce objective responses in patients with CMML, suggesting its potential as an effective treatment option.
Unplugging JAK/STAT in Chronic Myelomonocytic Leukemia.Solary, E.[2021]

Citations

Tretinoin Enhances the Effects of Chemotherapy in Juvenile Myelomonocytic Leukemia Using an Ex Vivo Drug Sensitivity Assay. [2023]
[Clinical features and prognosis of juvenile myelomonocytic leukemia: an analysis of 63 cases]. [2023]
Unplugging JAK/STAT in Chronic Myelomonocytic Leukemia. [2021]
Allogeneic bone marrow transplant improves outcome for juvenile myelomonocytic leukaemia. [2019]
Potential clinical use of azacitidine and MEK inhibitor combination therapy in PTPN11-mutated juvenile myelomonocytic leukemia. [2023]
Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: a report from the Children's Oncology Group. [2018]
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