Phase I Trial of Ziftomenib Combined With Venetoclax and Azacitidine for Children and Young Adults With Relapsed or Refractory Acute Leukemia Having Specific Genetic Mutations

What this Trial Is About

Researchers are studying the safety and best dose of ziftomenib combined with venetoclax and azacitidine in children and young adults aged 2 to 21 years with relapsed or refractory acute leukemias that have specific genetic mutations such as KMT2A-r, NPM1-m, NUP98-r, or HOX pathway changes. This Phase I trial aims to find the highest dose of ziftomenib that can be safely given with the other two drugs, while also evaluating early signs of how well this combination works and exploring molecular markers related to the treatment. Participants will receive ziftomenib intravenously along with oral venetoclax and azacitidine. The study begins with a small group receiving the starting dose of ziftomenib if no severe side effects occur, later participants will get higher doses. If side effects occur, lower doses or shorter treatment times will be tested. This dose-escalation approach helps determine the recommended dose for future studies. During the study, participants will be closely monitored for safety and side effects for about one year. Researchers will assess treatment responses, including complete remission and survival outcomes. Various tests including blood counts, heart function, liver and kidney function, and molecular studies will be performed regularly. Participants health will be followed to understand the treatments effects and any resistance that may develop.

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