Experimental leukemia drug made available for patients who have no other options

NCT ID NCT05738538

First seen Jun 29, 2026 · Last updated Jun 30, 2026 · Updated 1 time

Summary

This program provides expanded access to the experimental drug ziftomenib for adults with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that has specific genetic changes (KMT2A rearrangements or NPM1 mutations). It is for patients who have exhausted standard treatments and have no satisfactory alternatives. The drug is given alone and is only available in the United States.

What this could mean

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

Active substance

ziftomenib

What this could lead to

If it works, this could provide a treatment option for people with hard-to-treat leukemias who have run out of standard therapies.

What could go wrong

This is an early-stage expanded access program, not a large clinical trial. The drug may not work for everyone and could have side effects.

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 acute myeloid leukemia with mutated NPM1 acute myeloid leukemia, MLL gene rearrangement B-lymphoblastic leukemia/lymphoma with t(v;11q23.3) Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

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