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ID06782971

Study of Venetoclax and Azacitidine Treatment Effects in Adult AML Using Patient-Derived Mouse Models

Led by Clinical Hub for Interventional Research (CHOIR) · Updated on 2025-12-08

25

Participants Needed

4

Research Sites

108 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are investigating new ways to test drug combinations aimed at killing tumor cells in patients with acute myeloid leukemia AML. This observational study focuses on whether using AML cells from individual patients in special mice that can accept human tissue can speed up the discovery of better treatments. It also evaluates if these mice show treatment responses similar to the actual AML patients from whom the cells were taken. Participants in this study are individuals with AML who are receiving the standard treatment of venetoclax and azacitidine. Blood and bone marrow samples will be collected from these patients to create patient-derived xenografts PDX in mice. These mice models mimic the patients disease and treatment response, allowing researchers to test standard and multiple experimental therapies simultaneously over a period of up to two years. The study aims to generate at least 20 adult AML PDX models linked with detailed clinical data. During the study, participants will provide samples before and after starting venetoclax plus azacitidine therapy. Researchers will measure leukemic cell frequency and characteristics using flow cytometry in both the mouse models and patient samples. The main outcomes include the successful creation of AML PDX models and the evaluation of their treatment responses over up to 52 weeks. This approach supports precision therapies tailored to specific tumor profiles and may accelerate future clinical trials of promising drug combinations.

CONDITIONS

Brief Title

Evaluation of Combined Sensitising and Hypomethylating Therapy Outcomes in AML PDX

Research Team

J

John Pimanda, Professor

M

Mark Polizzotto, Professor

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