NCT06448013

A Phase I Study Investigating the Combination of the Menin Inhibitor Ziftomenib With Venetoclax and Gemtuzumab in Pediatric Patients With Acute Myeloid Leukemia

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 22 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 3 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

To find the recommended dose of ziftomenib in combination with gemtuzumab ozogamicin and venetoclax that can be given to pediatric participants who have relapsed or refractory AML or MPAL.

Detailed description

Primary Objectives: \- To determine the safety, tolerability, and recommended Phase II dose (RP2D) of ziftomenib in combination with gemtuzumab ozogamicin and venetoclax for pediatric participants with acute myeloid leukemia (AML). Secondary Objectives: * To determine the preliminary assessment of efficacy by overall response (OR), including complete remission (CR), CR with incomplete blood count recovery and partial remission, overall survival (OS), event-free survival (EFS) and duration of response (DOR) of pediatric participants treated with this combination. * To determine time to first response and time to best response of pediatric participants treated with this combination. Exploratory Objective \- To evaluate molecular and cellular markers that may be predictive of antitumor activity and/or resistance.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Venetoclax	Given by PO
DRUG	Gemtuzumab	Given by IV
DRUG	Ziftomenib	Given by PO

Timeline

• Start date: 2025-03-07
• Primary completion: 2028-12-31
• Completion: 2030-12-31
• First posted: 2024-06-07
• Last updated: 2025-12-15

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study