New pill shows promise for Tough-to-Treat leukemias
NCT ID NCT02392572
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests an oral drug called ONC201 in people whose acute leukemia or high-risk myelodysplastic syndrome (MDS) has returned or not responded to standard treatments. The trial has two phases: Phase I finds the safest dose, and Phase II checks if the drug shrinks or controls the cancer. About 120 adults will take part, and some may also receive a second drug called venetoclax.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ONC201 (a drug that may block enzymes needed for cancer cell growth, sometimes given with venetoclax)
What this could lead to
If it works, this could offer a new treatment option for people with leukemia or MDS that has come back or stopped responding to other therapies.
What could go wrong
This is an early-phase trial (Phase I/II) with only 120 participants, so the drug may not prove effective or may have side effects. It is not yet known 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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M D Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-••••
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