Trametinib for Juvenile Myelomonocytic Leukemia

Recruiting in Palo Alto (17 mi)

+57 other locations

Overseen ByElliot Stieglitz

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: National Cancer Institute (NCI)

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Approved in 4 jurisdictions

Trial Summary

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.

Eligibility Criteria

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.

I can swallow pills or use a feeding tube.

I have recovered from side effects of my previous cancer treatments.

Exclusion Criteria

I am taking medication to prevent organ rejection after a transplant.

I have had a liver condition called sinusoid obstructive syndrome in the last 3 months.

I have a history or risk of eye vein blockage or swelling.

My chronic conditions like high blood pressure or diabetes are under control.

I do not have any infections that are currently uncontrolled.

I have been diagnosed with Noonan syndrome.

I am not on any cancer treatments except possibly hydroxyurea.

Participant Groups

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.

1Treatment groups

Experimental Treatment

Group I: Treatment (trametinib)Experimental Treatment2 Interventions

Patients receive trametinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo a bone marrow aspiration or biopsy at baseline, on day 28 of cycles 1 and 2, at all subsequent odd numbered cycles, and at end of treatment.

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

🇪🇺 Approved in European Union as Mekinist for: