Promising combo for tough leukemia hits roadblock: trial stopped early

NCT ID NCT04874194

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-phase trial tested a combination of two drugs, omacetaxine and venetoclax, in 24 adults with relapsed or refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that had a specific genetic change called RUNX1. The goal was to find the best dose and see if the combo could help control the disease. However, the study was terminated early, so we don't have full results on how well it worked.

What this could mean

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

Active substance

Omacetaxine and venetoclax

What this could lead to

If it had worked, this combination could have offered a new treatment option for people with hard-to-treat leukemia or MDS who have a specific genetic change (RUNX1 mutation).

What could go wrong

The trial was terminated early, so we don't have clear evidence that this combination works. It was also a small, early-phase study, so even if it had continued, success was not guaranteed.

Disclaimer Read more

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.

acute biphenotypic leukemia acute myeloid leukemia hematopoietic and lymphoid cell neoplasm hematopoietic and lymphoid system neoplasm myelodysplastic syndrome Myelodysplastic Syndromes

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