New hope for kids with tough leukemia: targeted drug combo enters first human trial
NCT ID NCT06376162
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial tests a new drug called ziftomenib, taken as a capsule, together with standard chemotherapy (cytarabine and fludarabine) for children and young adults up to age 21 who have a relapsed or refractory acute leukemia with specific genetic changes (KMT2A-r, NUP98-r, or NPM1-m). The main goal is to find the safest dose and understand how the drug behaves in the body. Only 20 participants will be enrolled, and the study is currently recruiting.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ziftomenib (a menin inhibitor) combined with chemotherapy (cytarabine and fludarabine)
What this could lead to
If successful, this could point toward a new treatment option for children with hard-to-treat leukemia that has come back or not responded to standard therapy.
What could go wrong
This is a very early phase 1 trial with only 20 participants, so safety and dosing are still being figured out. It may not work or could cause serious side effects.
Disclaimer
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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.
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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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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CHU de Nantes - Hôpital Femme-Enfant-Adolescent
RECRUITINGNantes, Loire-Atlantique, 44093, France
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CHU de Reims - Hôpital Robert Debré
RECRUITINGParis, Île-de-France Region, 75019, France
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Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229-3026, United States
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Dana-Farber Cancer Institute
RECRUITINGBoston, Massachusetts, 02215, United States
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Fondazione IRCCS San Gerardo dei Tintori (Ospedale San Gerardo)
RECRUITINGMonza, 20900, Italy
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Hospital Infantil Universitario Niño Jesús
RECRUITINGMadrid, 28009, Spain
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Hospital Universitari Vall d'Hebrón
RECRUITINGBarcelona, 08035, Spain
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Memorial Sloan Kettering Cancer Center - New York
RECRUITINGNew York, New York, 10065, United States
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Ospedale Pediatrico Bambino Gesù
RECRUITINGRoma, Rome, 00165, Italy
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Prinses Maxima Centrum Kinderoncologie
RECRUITINGUtrecht, 3584 CS, Netherlands
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Sankt Anna-Kinderspital
RECRUITINGVienna, State of Vienna, 1090, Austria
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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SickKids - The Hospital for Sick Children
RECRUITINGToronto, Ontario, M5G 1X8, Canada
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St. Jude Children's Research Hospital
RECRUITINGMemphis, Tennessee, 38105-3678, United States
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Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States