New hope for kids with tough leukemias: experimental combo trial opens

NCT ID NCT06397027

Recruiting now Disease control Sponsor: M.D. Anderson Cancer Center Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 1 study is testing a combination of three drugs—ziftomenib, venetoclax, and azacitidine—in children and young adults (ages 2 to 21) with acute leukemia that has returned or not responded to treatment. The main goal is to find the safest dose of ziftomenib when given with the other two drugs. The trial enrolls 22 participants whose leukemia has specific genetic mutations. It is currently recruiting at M.D. Anderson Cancer Center.

What this could mean

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

Active substance: ziftomenib, venetoclax, and azacitidine
What this could lead to: If successful, this could lead to a new treatment option for children with hard-to-treat acute leukemias that have specific genetic changes.
What could go wrong: This is a very early phase 1 trial with only 22 participants, so it is primarily testing safety and dosing. The combination may not work or could cause serious side effects.

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Conditions

The condition(s) this trial relates to.

acute leukemia acute leukemia of ambiguous lineage acute lymphoblastic leukemia acute myeloid leukemia mixed phenotype acute leukemia

As listed by the trial registrant

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

PEDIATRIC RELAPSED REFRACTORY ACUTE LEUKEMIA

Contacts and locations

Locations

MD Anderson Cancer Center
RECRUITING

Houston, Texas, 77030, United States

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