New drug combo aims to tame rare childhood leukemia
NCT ID NCT05849662
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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C.S. Mott Children's Hospital
RECRUITINGAnn Arbor, Michigan, 48109, United States
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 900027, United States
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Children's Hospital of Atlanta
RECRUITINGAtlanta, Georgia, 30322, United States
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Children's Hospital of Colorado
RECRUITINGDenver, Colorado, 80045, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Mercy Hospital
RECRUITINGKansas City, Missouri, 64108, United States
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Children's National Medical Center
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
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Indiana University/Riley Hospital for Children
RECRUITINGIndianapolis, Indiana, 46202, United States
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Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Memorial Sloan Kettering Cancer Center
RECRUITINGNew York, New York, 10065, United States
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Oregon Health & Science University
RECRUITINGPortland, Oregon, 97239, United States
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Phoenix Children's Hospital
RECRUITINGPhoenix, Arizona, 85016, United States
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Primary Children's Hospital
RECRUITINGSalt Lake City, Utah, 84113, United States
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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Sidney Kimmel Cancer Center at Johns Hopkins
RECRUITINGBaltimore, Maryland, 21231, United States
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St. Jude Children's Research Hospital Memphis
RECRUITINGMemphis, Tennessee, 38105, United States
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University of California San Francisco
RECRUITINGSan Francisco, California, 94158, United States
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University of Miami
RECRUITINGMiami, Florida, 33136, United States
Contact