New hope for kids with tough leukemia: oral drug combo enters human testing

NCT ID NCT06191978

Recruiting now Disease control Sponsor: M.D. Anderson Cancer Center Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial is testing a combination of two oral drugs—venetoclax and decitabine/cedazuridine—in children and teens whose acute myeloid leukemia (AML) has returned or not responded to prior treatment. The study aims to find the safest dose and check for early signs that the drugs are working. Up to 40 participants will be enrolled at MD Anderson Cancer Center.

What this could mean

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

Active substance: venetoclax and decitabine/cedazuridine (ASTX727)
What this could lead to: If successful, this could lead to a new oral combination therapy for children with hard-to-treat AML that has come back or not responded to standard treatment.
What could go wrong: This is an early Phase 1 trial with only 40 participants, so the main goal is safety and dosing, not proof of effectiveness. The combination may cause side effects or fail to control the leukemia long-term.

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

As listed by the trial registrant

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

ACUTE MYELOID LEUKEMIA

Contacts and locations

Locations

MD Anderson Cancer Center
RECRUITING

Houston, Texas, 77030, United States

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