Actively Recruiting
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
Led by Therapeutic Advances in Childhood Leukemia Consortium · Updated on 2026-04-15
58
Participants Needed
19
Research Sites
268 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be between 1 month and 21 years of age at enrollment.
- Patients must meet the 2022 International Consensus Classification criteria for JMML, including specific clinical and genetic features.
- Karnofsky performance status greater than 50% for patients aged 16 years or older.
- Lansky performance status greater than 50% for patients younger than 16 years.
- No prior leukemia-directed therapy except limited cytoreduction with hydroxyurea or 6-mercaptopurine before starting trametinib.
- No prior hematopoietic stem cell transplant.
- Adequate renal function defined by creatinine clearance, glomerular filtration rate, or normal serum creatinine for age and gender.
- Adequate liver function with direct bilirubin less than 1.5 times the upper limit of normal and ALT less than 5 times the upper limit.
- Adequate cardiac function with ejection fraction of 50% or higher by echocardiogram or radionuclide angiogram.
- Female patients of childbearing potential must have a negative pregnancy test within 2 weeks before enrollment.
- Female patients with infants must agree not to breastfeed during the study.
- Male and female patients of childbearing potential must agree to use effective contraception during the study and for 6 months after treatment.
You will not qualify if you...
- Known allergy to any of the study drugs (trametinib, azacitidine, fludarabine, cytarabine).
- Active systemic infection with ongoing symptoms despite appropriate treatment; patient must be off pressors and have negative blood cultures for 48 hours.
- Planned use of non-protocol chemotherapy, radiation, or immunotherapy during the study.
- Significant concurrent illness, psychiatric disorder, or social issue that may affect safety or compliance.
- Clinical or molecular diagnosis of Noonan syndrome.
- Prior use of hematopoietic growth factors, biologics, or radiation therapy.
- Taking medications for treatment of left ventricular systolic dysfunction.
- History or current evidence of retinal vein occlusion or central serous retinopathy.
- Prior use of any MEK inhibitor.
- Diagnosis of Down syndrome.
AI-Screening
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Trial Site Locations
Total: 19 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 900027
Actively Recruiting
3
University of California San Francisco
San Francisco, California, United States, 94158
Actively Recruiting
4
Children's Hospital of Colorado
Denver, Colorado, United States, 80045
Actively Recruiting
5
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
7
Children's Hospital of Atlanta
Atlanta, Georgia, United States, 30322
Actively Recruiting
8
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
9
Indiana University/Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Actively Recruiting
10
Sidney Kimmel Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231
Actively Recruiting
11
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
12
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Actively Recruiting
13
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
14
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
15
Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
16
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
17
St. Jude Children's Research Hospital Memphis
Memphis, Tennessee, United States, 38105
Actively Recruiting
18
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Actively Recruiting
19
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
Research Team
E
Ellynore Florendo
CONTACT
S
Suganya Rajendran
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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