New drug combo aims to tame rare childhood leukemia

NCT ID NCT05849662

Recruiting now Disease control Sponsor: Therapeutic Advances in Childhood Leukemia Consortium Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tests a combination of two drugs, trametinib and azacitidine, for children newly diagnosed with juvenile myelomonocytic leukemia (JMML), a rare blood cancer. Lower-risk patients get just these two drugs, while higher-risk patients also receive standard chemotherapy. The goal is to see if this approach is safe and works better than current treatments. About 58 children and young adults up to age 21 will take part.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: trametinib and azacitidine (with or without fludarabine and cytarabine)
What this could lead to: If successful, this could provide a more effective, risk-adapted treatment for children with JMML, potentially improving survival and reducing side effects.
What could go wrong: This is an early-phase trial with only 58 participants, so results may not apply to all patients. The drug combinations can cause serious side effects like infections and organ damage.

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Conditions

The condition(s) this trial relates to.

CBL-related disorder juvenile myelomonocytic leukemia neurofibromatosis type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CBL SYNDROME JCML JMML LEUKEMIA, JUVENILE MYELOMONOCYTIC NEUROFIBROMATOSIS 1

Contacts and locations

Locations

C.S. Mott Children's Hospital
RECRUITING

Ann Arbor, Michigan, 48109, United States
Children's Hospital Los Angeles
RECRUITING

Los Angeles, California, 900027, United States
Children's Hospital of Atlanta
RECRUITING

Atlanta, Georgia, 30322, United States
Children's Hospital of Colorado
RECRUITING

Denver, Colorado, 80045, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's Mercy Hospital
RECRUITING

Kansas City, Missouri, 64108, United States
Children's National Medical Center
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Indiana University/Riley Hospital for Children
RECRUITING

Indianapolis, Indiana, 46202, United States
Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Memorial Sloan Kettering Cancer Center
RECRUITING

New York, New York, 10065, United States
Oregon Health & Science University
RECRUITING

Portland, Oregon, 97239, United States
Phoenix Children's Hospital
RECRUITING

Phoenix, Arizona, 85016, United States
Primary Children's Hospital
RECRUITING

Salt Lake City, Utah, 84113, United States
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
Sidney Kimmel Cancer Center at Johns Hopkins
RECRUITING

Baltimore, Maryland, 21231, United States
St. Jude Children's Research Hospital Memphis
RECRUITING

Memphis, Tennessee, 38105, United States
University of California San Francisco
RECRUITING

San Francisco, California, 94158, United States
University of Miami
RECRUITING

Miami, Florida, 33136, United States

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