Promising combo for IDH1 leukemia hits early snag – trial stopped

NCT ID NCT04250051

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

Summary

This early-phase trial tested whether adding the targeted drug ivosidenib to standard FLAG chemotherapy could help people with a specific genetic form of acute myeloid leukemia (IDH1-mutant AML). The study aimed to find the best dose and check for side effects in patients whose cancer was newly diagnosed, had returned, or was not responding to treatment. However, the trial was terminated early after enrolling only 2 participants, so we have very limited information about its safety or effectiveness.

What this could mean

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

Active substance

ivosidenib

What this could lead to

If it works, this could point toward a more effective treatment for people with IDH1-mutant AML that has returned or not responded to therapy.

What could go wrong

This was a very early, small Phase 1 trial that was terminated after enrolling only 2 participants. The combination may cause serious side effects, and it is unknown if it works better than standard care.

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 myeloid leukemia myelodysplastic syndrome Myelodysplastic Syndromes myelodysplastic/myeloproliferative neoplasm myeloid neoplasm Myeloproliferative Disorders myeloproliferative neoplasm

As listed by the trial registrant

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

Contacts and locations

Locations

  • Northwestern University

    Chicago, Illinois, 60611, United States