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Nucleoside Analog

Trametinib + Azacitidine for Myelomonocytic Leukemia

Phase 1 & 2
Recruiting
Research Sponsored by Therapeutic Advances in Childhood Leukemia Consortium
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Karnofsky performance level > 50% for patients ≥ 16 years of age, Lansky performance level > 50% for patients < 16 years of age
Patients must be ≥ 1 month and ≤21 years of age at enrollment
Must not have
Clinical or molecular diagnosis of Noonan syndrome
Prior use of hematopoietic growth factors, biologics, or XRT
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at the end of the evaluation period of cycle 1 (defined as 28 day cycle of therapy plus 30 days following the last dose of study therapy)
Awards & highlights

Summary

This trial will test if combining trametinib and azacitidine is safe and effective for treating juvenile myelomonocytic leukemia (JMML). Lower-risk patients will receive trametin

Who is the study for?
This trial is for children and young adults up to 21 years old with newly diagnosed juvenile myelomonocytic leukemia (JMML). Participants must meet specific diagnostic criteria, including spleen enlargement, high monocyte counts, and no BCR/ABL fusion gene. They should also have a RAS mutation or clinical diagnosis of neurofibromatosis type 1.
What is being tested?
The trial is testing the combination of trametinib and azacitidine in lower-risk JMML patients. High-risk patients will additionally receive fludarabine and cytarabine. It aims to determine how safe this treatment is and how effective it is at treating the disease.
What are the potential side effects?
Potential side effects may include skin rash, diarrhea, fatigue, nausea, liver enzyme changes for trametinib; low blood cell counts, infection risk increase for azacitidine; mouth sores and immune system suppression for fludarabine; fever with low white blood cells count for cytarabine.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can do most activities by myself if I am 16 or older.
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I am between 1 month and 21 years old.
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My kidney function is normal for my age and gender.
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My heart pumps blood well according to my last test.
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I am not pregnant, will not get pregnant, and will not breastfeed during the trial.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have been diagnosed with Noonan syndrome.
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I have previously used growth factors, biologics, or radiation therapy.
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I have previously used a MEK inhibitor.
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I have an infection that hasn't improved with treatment.
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I have had retinal vein occlusion or central serous retinopathy.
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I plan to receive other cancer treatments not listed in the study during the trial.
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I am on medication for heart muscle weakness.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at the end of the evaluation period of cycle 1 (defined as 28 day cycle of therapy plus 30 days following the last dose of study therapy)
This trial's timeline: 3 weeks for screening, Varies for treatment, and at the end of the evaluation period of cycle 1 (defined as 28 day cycle of therapy plus 30 days following the last dose of study therapy) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To determine the safety of combining trametinib with azacitidine (Aza), fludarabine (FLA) and cytarabine for patients with newly diagnosed high-risk JMML.
To determine the safety of combining trametinib with azacitidine for patients with newly diagnosed lower-risk JMML.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Lower-risk patientsExperimental Treatment2 Interventions
Lower-risk patients are defined as having a mutational burden of one clonal alteration AND a low DNA methylation classification.
Group II: High-risk patientsExperimental Treatment4 Interventions
High-risk patients are defined as having as having a mutational burden of more than one clonal alteration AND/OR an intermediate or high DNA methylation classification.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Azacitidine
2012
Completed Phase 3
~1440
Fludarabine
2012
Completed Phase 4
~1860
Trametinib
2014
Completed Phase 2
~1550
Cytarabine
2016
Completed Phase 3
~3330

Find a Location

Who is running the clinical trial?

Therapeutic Advances in Childhood Leukemia ConsortiumLead Sponsor
20 Previous Clinical Trials
619 Total Patients Enrolled
~39 spots leftby Dec 2028
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