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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

No Placebo-Only Group

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 weeks3 visits

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)

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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.

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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