New drug combo aims to keep childhood leukemia at bay after transplant

NCT ID NCT07012044

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

Summary

This phase 1 trial tests the safety and best dose of an oral drug called ASTX727 (a combination of cedazuridine and decitabine) along with filgrastim in children with high-risk acute myeloid leukemia (AML) that has returned or not responded to treatment. All participants have already received a stem cell transplant. The goal is to see if this maintenance therapy can help prevent the leukemia from coming back without causing severe side effects.

What this could mean

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

Active substance

ASTX727 (cedazuridine and decitabine) and filgrastim

What this could lead to

If it works, this could provide a safer maintenance option after stem cell transplant to help prevent leukemia relapse in children.

What could go wrong

This is an early phase 1 trial with only 47 participants, so safety and dosing are still being determined. It may not work or could cause serious side effects like graft-versus-host disease.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for RECURRENT ACUTE MYELOID LEUKEMIA are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

acute myeloid leukemia therapy related acute myeloid leukemia and myelodysplastic syndrome

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's Hospital of Alabama

    RECRUITING

    Birmingham, Alabama, 35233, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Hospital of Pittsburgh of UPMC

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Memorial Sloan Kettering Cancer Center

    RECRUITING

    New York, New York, 10065, United States

    Contact

    Contact Phone: •••-•••-••••

  • Riley Hospital for Children

    RECRUITING

    Indianapolis, Indiana, 46202, United States

    Contact

    Contact Phone: •••-•••-••••

  • Saint Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of Minnesota/Masonic Cancer Center

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

    Contact

    Contact Phone: •••-•••-••••