CHIP-AML22 Master Protocol A Phase 3 Trial to Improve Treatment and Reduce Side Effects in Children and Adolescents with Newly Diagnosed de Novo Acute Myeloid Leukemia

What this Trial Is About

Researchers are evaluating new treatment approaches for children and adolescents newly diagnosed with de novo acute myeloid leukemia AML to improve their chances of event-free survival. The study aims to increase cure rates while minimizing unnecessary toxicity by comparing different chemotherapy regimens and adding targeted therapies. This phase 3 master protocol trial also explores risk-adapted treatment based on genetic markers and disease response, as well as cardioprotective measures and quality of life during and after treatment. Participants receive chemotherapy courses with different consolidation strategies, with some patients randomized to receive either three or two consolidation chemotherapy cycles. Additionally, for CD33-positive patients, the study compares adding gemtuzumab ozogamicin to the first induction chemotherapy course versus not adding it. Patients with certain genetic profiles may receive the FLT3 inhibitor quizartinib alongside chemotherapy and as continuation therapy after stem cell transplant if disease markers remain. The study involves stratification of patients into risk groups to tailor treatment intensity and includes use of a heart-protecting drug during specific chemotherapy courses. During the trial, participants will undergo diagnostic tests including flow cytometry and genetic analysis to guide treatment allocation. Researchers will monitor event-free survival over five years and assess treatment toxicity, safety, healthcare resource use, and quality of life through questionnaires. Frequent follow-up visits will evaluate disease status and side effects. The trial plans long-term observation to understand effectiveness and safety of the strategies tested, and participation requires compliance with follow-up and management of side effects throughout treatment and beyond.

CHIP-AML22/Master: An Open Label Complex Clinical Trial in Newly Diagnosed Pediatric de Novo AML Patients