Phase III Study of Trametinib and Azacitidine With or Without Chemotherapy for Newly Diagnosed Juvenile Myelomonocytic Leukemia

What this Trial Is About

Researchers are evaluating the safety and effectiveness of combining trametinib and azacitidine in children and young adults newly diagnosed with juvenile myelomonocytic leukemia JMML. This study focuses on two groups lower-risk patients and high-risk patients, defined by specific genetic and molecular features. The trial aims to determine how well these drug combinations work and their safety in treating JMML. Lower-risk patients will receive daily azacitidine for five days combined with daily trametinib for 28 days per treatment cycle, for up to twelve cycles. High-risk patients will receive a combination of azacitidine, fludarabine, and cytarabine for five days along with daily trametinib for 28 days per cycle, for up to two cycles. These treatments are given through oral and intravenous methods, depending on the drug, and are tailored to the patients risk category. Participants will undergo evaluations to monitor safety and treatment effects during and after each treatment cycle. Assessments include blood tests, genetic studies, kidney, liver, and heart function checks, and monitoring for side effects. The study will measure safety outcomes at the end of the first treatment cycle plus 30 days. Patients are followed throughout treatment courses lasting up to twelve cycles for lower-risk or two cycles for high-risk JMML, with ongoing monitoring to ensure their well-being.

A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia