Promising combo for relapsed leukemia in kids hits early hurdle

NCT ID NCT04240002

Terminated Disease control Sponsor: Astellas Pharma Global Development, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called gilteritinib, which targets a specific genetic change in leukemia cells, combined with standard chemotherapy in children and young adults (ages 6 months to 21 years) whose acute myeloid leukemia (AML) had returned or not responded to treatment. The goal was to find the best dose and see if the combination could lead to remission. Only 9 people took part, and the study was stopped early, so we have limited information on how well it works.

What this could mean

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

Active substance: gilteritinib (a targeted drug) combined with chemotherapy drugs fludarabine, cytarabine, and G-CSF
What this could lead to: If successful, this combination could offer a new treatment option for young patients with a hard-to-treat form of leukemia that has come back or not responded to standard therapy.
What could go wrong: This was a very early, small study (only 9 participants) that was terminated, so results are limited. The combination may cause severe side effects, and it is unclear if it works better than existing treatments.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia acute myeloid leukemia, FLT3 internal tandem duplication

As listed by the trial registrant

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

ACUTE MYELOID LEUKEMIA (AML) ACUTE MYELOID LEUKEMIA WITH FMS-LIKE TYROSINE KINASE 3 (FLT3) MUTATION / INTERNAL TANDEM DUPLICATION (ITD)

Contacts and locations

Locations

SIte IT39001

Roma, 165, Italy
Site DE49004

Essen, North Rhine-Westphalia, 45147, Germany
Site ES34001

Barcelona, 08950, Spain
Site GB44001

Birmingham, B4 6NH, United Kingdom
Site GB44005

Cardiff, CF14 4XW, United Kingdom
Site UK44007

Sutton, United Kingdom
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

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