RecruitingPhase 1Phase 2NCT05849662

A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Risk Stratified Treatment for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia: A Phase I/II Non-randomized Study of Trametinib and Azacitidine With or Without Chemotherapy (IND #164058)


Sponsor

Therapeutic Advances in Childhood Leukemia Consortium

Enrollment

58 participants

Start Date

Oct 11, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML). Newly diagnosed lower-risk JMML patients will receive trametinib and azacitidine. High-risk JMML patients will receive trametinib, azacitidine, fludarabine, and cytarabine.


Eligibility

Min Age: 1 MonthMax Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a combination of two drugs — trametinib (which blocks abnormal cell growth signals) and azacitidine (a chemotherapy that affects how genes are expressed) — for children and young people newly diagnosed with juvenile myelomonocytic leukemia (JMML), a rare and serious blood cancer in young children. **You may be eligible if...** - You are between 1 month and 21 years old at enrollment - You have a newly confirmed diagnosis of JMML based on specific diagnostic criteria (including certain gene mutations and blood test results) - You have not yet received treatment for JMML (other than basic supportive care) **You may NOT be eligible if...** - You have already received targeted therapy or chemotherapy for JMML - You have serious organ damage or active infections - You have a history of certain other genetic syndromes that would exclude participation - You are pregnant or breastfeeding (for participants of childbearing potential) Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGTrametinib

PO or NG QD Days 1-28 For patients age \< 6 years: 0.032 mg/kg/day at max dose = 2mg/day For patients age ≥ 6 years: 0.025 mg/kg/day at max dose = 2 mg/day

DRUGAzacitidine

IV over 30 minutes Days 1-5 Age \< 1 year or weight \<10kg: 2.5 mg/kg/day Age ≥ 1 year and weight ≥ 10kg: 75 mg/m2/day

DRUGFludarabine

IV over 30 minutes Days 6-10 30 mg/m2/day (1mg/kg if \<12 kg)

DRUGCytarabine

IV over 3 hours Days 6-10 2000 mg/m2/day (67mg/kg if \<12 kg)


Locations(19)

Phoenix Children's Hospital

Phoenix, Arizona, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

University of California San Francisco

San Francisco, California, United States

Children's Hospital of Colorado

Denver, Colorado, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

University of Miami

Miami, Florida, United States

Children's Hospital of Atlanta

Atlanta, Georgia, United States

Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Indiana University/Riley Hospital for Children

Indianapolis, Indiana, United States

Sidney Kimmel Cancer Center at Johns Hopkins

Baltimore, Maryland, United States

C.S. Mott Children's Hospital

Ann Arbor, Michigan, United States

Children's Mercy Hospital

Kansas City, Missouri, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

St. Jude Children's Research Hospital Memphis

Memphis, Tennessee, United States

Primary Children's Hospital

Salt Lake City, Utah, United States

Seattle Children's Hospital

Seattle, Washington, United States

View Full Details on ClinicalTrials.gov

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NCT05849662


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