Promising combo for relapsed leukemia in kids hits early hurdle
NCT ID NCT04240002
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.
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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Locations
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SIte IT39001
Roma, 165, Italy
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Site DE49004
Essen, North Rhine-Westphalia, 45147, Germany
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Site ES34001
Barcelona, 08950, Spain
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Site GB44001
Birmingham, B4 6NH, United Kingdom
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Site GB44005
Cardiff, CF14 4XW, United Kingdom
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Site UK44007
Sutton, United Kingdom
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States