NCT07355335

Ziftomenib + Mezigdomide in Adolesc. and Adults w/ R/R AML

A Phase 1 Study of Menin-KMT2A Inhibitor Ziftomenib (KO-539) in Combination With Cereblon E3 Ligase Modulator Mezigdomide (CC-92480) in Adolescents and Adults With Relapsed or Refractory Acute Myeloid Leukemia (AML)

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Massachusetts General Hospital · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and tolerability of mezigdomide in combination with ziftomenib in adolescent and adult participants with either KMT2A-rearranged (KMT2A-r) or NPM1-mutant relapsed or refractory acute myeloid leukemia (AML).

Detailed description

This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the appropriate dose of the investigational drug to use for further studies. "Investigational" means that the drug is being studied. The U.S. Food and Drug Administration (FDA) has not approved ziftomenib and mezigdomide as a treatment for any disease. Duration of treatment will depend on individual response, evidence of disease progression, and tolerance. The total treatment period will be 12 cycles. If you have completed treatment, you will be followed of up to 12 months. If you do not, complete treatment, you will be followed for 30 days after discontinuation of treatment. It is expected that about 24 people will take part in this research study.

Conditions

• KMT2A-rearranged
• NPM1-mutant Refractory or Relapsed AML

Interventions

Type	Name	Description
DRUG	Ziftomenib	Capsule taken orally once daily on days 1-28 of each 28-day cycle.
DRUG	Mezigdomide	Capsule taken orally once daily on days 1-21 or possibly days 1-14 of each 28-day cycle.

Timeline

• Start date: 2026-07-09
• Primary completion: 2027-07-01
• Completion: 2028-01-01
• First posted: 2026-01-21
• Last updated: 2026-01-22

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study