New hope for kids with tough leukemia: oral drug combo enters human testing
NCT ID NCT06191978
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 30 times
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.
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This is a summary of
the original study
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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MD Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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.
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.