Testing a new leukemia medication in children who haven't responded to other drugs

Plain-English translation of NCT04925479 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated July 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This study is testing a medication called asciminib in children and teenagers (ages 1–18) who have chronic myeloid leukemia and haven't responded well to previous treatments. The trial aims to figure out the right dose of this medication for different ages and to understand how the medication works in young patients' bodies. Researchers want to make sure the treatment is safe and effective before it becomes widely available for children.

Most leukemia medications have been studied mainly in adults, and doctors need to know the right dose and safety profile for children. Some young patients stop responding to standard treatments, and this medication offers a new option for those who need it.

Do you qualify?

You likely qualify if…

you are between 1 and 18 years old (or 14–18 years old if receiving the adult-strength formulation)
you have been diagnosed with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase
you have already been treated with at least one other tyrosine kinase inhibitor medication
your leukemia has not responded well to your most recent treatment or you have had difficulty tolerating it
your blood and bone marrow meet specific criteria showing your leukemia is in a stable, early phase

You likely don't qualify if…

you have a specific genetic mutation (T315I) or other BCR-ABL mutations known to resist this treatment
you have had a stem cell transplant in the past or are planning to have one
you have heart rhythm problems or other serious heart conditions
you have had pancreatitis (pancreas inflammation) in the past year or have chronic pancreatitis
you are pregnant, breastfeeding, or have serious liver disease or problems absorbing food in your stomach

What participation looks like

You will take the medication by mouth twice a day (or once a day in later parts of the study) with food. The study is divided into stages: first, researchers will find the right dose for your age and weight, then more participants will be added to confirm the dose is safe and effective. You'll have regular clinic visits for blood tests, health checks, and to report any side effects. The total treatment period lasts as long as the medication is helping you and you're tolerating it well.

AI-generated summary from trial data · Jul 4, 2026 · Not medical advice

Trial locations(39 sites)

United States

Indiana UH Riley H for CIU, Indianapolis, Indiana

Dana Farber Cancer Institute, Boston, Massachusetts

University of Mississippi Medical Center, Jackson, Mississippi

Columbia University Medical Center New York Presbyterian, New York, New York

Cinn Children Hosp Medical Center, Cincinnati, Ohio