CHIP-AML22: clinical trial for newly diagnosed pediatric de novo AML patients

A new trial, CHIP-AML22, has been launched in Belgium in October 2024. CHIP-AML22 is a prospective, randomized, open-label, international multicentric trial which includes the European frontline treatment approach for children and adolescents with acute myeloid leukemia (AML).  This complex trial uses a stratified approach to assign patients to treatments, based on key biomarkers such as FLT3-ITD/NPM1wt alterations, CD33 expression, and treatment response. The protocol includes a refined risk-group adapted therapy (standard risk (SR) and high risk (HR)).

Since pediatric AML is rare, international collaborations are required to develop and evaluate new treatment strategies with sufficient statistical power to ultimately improve outcome. The CHIP-AML22 trial follows a standard of care backbone chemotherapy approach for pediatric AML patients. It includes MEC as a first induction course, followed by ADE as the second course. Patients will then undergo intensively timed courses, with high-risk (HR) patients proceeding to Hematopoietic Stem Cell Transplantation (HSCT).  Additionally, the trial includes two randomizations.

The first randomization question includes a phase III open label randomization at induction with gemtuzumab ozogamicin (GO; Mylotarg®) in CD33-positive newly diagnosed AML. Patients will be randomized in the first induction MEC course with or without GO. The experimental arm will thus mean adding GO to the MEC course, which may potentially benefit treatment efficiency.

The second randomization in the protocol is the consolidation randomization question, investigating two versus three consolidation courses for standard-risk patients to try avoiding unnecessary toxicity.

The CHIP-AML22 trial is also designed to accommodate future linked clinical trials, including the single-arm trial with Quizartinib for FLT3-ITD/NPM1wt patients.

The overarching goal of this protocol is to further improve event-free survival in children and adolescents with newly diagnosed AML by using innovative medicines and optimized treatment strategies. Ultimately, findings from this trial aim to shape the future standard of care for this patient population.

The international sponsor of CHIP-AML22 trial is the Princess Máxima Center for Pediatric Oncology at Utrecht, Netherlands. In Belgium, it is coordinated by the University Hospital of Ghent, with Prof. Dr. Barbara De Moerloose as the national coordinating investigator. Six Belgian pediatric hematology-oncology centers are participating: UZ Gent, UZ Leuven, CUSL, HUDERF, CHU Liège (site de la Citadelle), and CHC Montlégia. For Belgium, we expect to recruit 60 patients over a period of 6 years.

This trial is open in Belgium thanks to the financial support of EU 4 Health and Kom Op Tegen Kanker.