Phase I Study for Pediatric Relapsed/Refractory Acute Leukemias

This study is for children and young adults (ages 2-21) with acute leukemia that has come back or is not responding to treatment. It's looking for the safest and most effective dose of three medicines given together: ziftomenib (given intravenously, meaning into a vein), venetoclax (taken by mouth), and azacitidine (taken by mouth). These medicines are being tested in patients whose leukemia has specific genetic changes (KMT2A-r, NPM1-m, NUP98-r, or HOX pathway mutations). The main goal is to understand the safety and side effects of this combination. The study also aims to see how well the treatment works in shrinking the leukemia. This is a Phase I study, meaning it's an early-stage trial focused on finding the right dose.

Study design
This is a Phase I interventional study, which means participants will receive the study treatments. It plans to enroll 22 participants to find the safest dose of the drug combination.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Safety and side effects will be monitored throughout the study, which is expected to last an average of 1 year.

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NCT06397027

A Phase I Study Investigating the Combination of the Ziftomenib, Venetoclax and Azacitidine in Pediatric Relapsed and Refractory Acute Leukemias

Recruiting
PHASE1Ages 2–21InterventionalTreatment
M.D. Anderson Cancer Center
~22 participants
Updated 2026-01-29 on ClinicalTrials.gov
What's tested:ZiftomenibVenetoclaxAzacitidine

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety and adverse events (AEs)
Measured over Through study completion; an average of 1 year
Refractory Acute Leukemia
Pediatric Relapsed
1 sites across 1 states
Texas1
  • David McCall, MD · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

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  • Safety and adverse events (AEs)Through study completion; an average of 1 year

    Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0