New drug combination targets rare childhood leukemia that resists standard treatment

NCT ID NCT07686107

First seen Jul 07, 2026 · Last updated Jul 23, 2026 · Updated 3 times

Summary

This trial tests whether adding the drug revumenib to standard chemotherapy (fludarabine and cytarabine) can help children and young adults with a rare form of acute myeloid leukemia (AML) that has come back or not responded to treatment. The study enrolls up to 27 participants aged 30 days to 22 years who have a specific genetic change called NUP98 rearrangement. Participants receive up to two cycles of the drug combination, and some may also receive a stem cell transplant followed by revumenib alone. The main goal is to see how many patients achieve remission after treatment.

What this could mean

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

Active substance
revumenib (SNDX-5613) combined with fludarabine and cytarabine (FLA chemotherapy)
What this could lead to
If successful, this combination could offer a new treatment option for children with this hard-to-treat leukemia, potentially leading to remission and enabling stem cell transplant.
What could go wrong
This is an early-phase trial with only 24 participants, so results may not apply broadly. The combination may cause significant side effects, and the leukemia may still resist treatment.

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.

As listed by the trial registrant

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