NCT06397027

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 22 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

To find the highest safe dose of ziftomenib that can be combined with venetoclax and azacitidine in pediatric participants with acute leukemia that has certain types of genetic mutations (changes).

Detailed description

Primary Objectives - To determine the safety, tolerability, and recommended Phase II dose (RP2D) of ziftomenib in combination with venetoclax and azacitidine for pediatric participants with acute leukemias with KMT2A-r, NPM1-m, NUP98-r, or HOX pathway mutations. Secondary Objectives \- To determine the preliminary assessment of efficacy by overall response (OR), including complete remission (CR), CR with partial hematological recovery (CRh), CR with incomplete blood count recovery (CRi), morphological leukemia-free state (MLFS) and partial remission (PR), overall survival (OS), event-free survival (EFS) and duration of response (DOR) 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. * To investigate relationships between PK/exposure and clinical outcomes (e.g.,safety/tolerability, efficacy).

Conditions

• Refractory Acute Leukemia
• Pediatric Relapsed

Interventions

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

Timeline

• Start date: 2024-12-27
• Primary completion: 2028-12-31
• Completion: 2030-12-31
• First posted: 2024-05-02
• Last updated: 2026-01-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study