New pill shows promise for Tough-to-Treat leukemias
NCT ID NCT02392572
First seen Jun 26, 2026 · Last updated Aug 06, 2026 · Updated 3 times
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.
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
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
M D Anderson Cancer Center
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Promising combo for IDH1 leukemia hits early snag – trial stopped