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New pill shows promise for tough blood cancers in early trial

NCT ID NCT02392572

First seen Nov 01, 2025 · Last updated Jun 05, 2026 · Updated 24 times

Summary

This study tests an oral drug called ONC201 in 120 adults whose acute leukemia or high-risk myelodysplastic syndrome (MDS) has returned or stopped responding to standard treatments. The goal is to find the safest dose and see if the drug can shrink or control the cancer. ONC201 works by blocking certain enzymes that cancer cells need to grow.

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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.

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Contacts and locations

Locations

  • M D Anderson Cancer Center

    RECRUITING

    Houston, Texas, 77030, United States

    Contact Phone: •••-•••-••••

    Contact

Conditions

The condition(s) this trial relates to.

acute lymphoblastic leukemia acute myeloid leukemia myelodysplastic syndrome Myelodysplastic Syndromes Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

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