A Phase 3 Study Comparing Intensive Chemotherapy With or Without Venetoclax in Children and Young Adults With Relapsed Acute Myeloid Leukemia

What this Trial Is About

Researchers are evaluating whether adding venetoclax to a chemotherapy combination of fludarabine, cytarabine, and gemtuzumab ozogamicin improves survival in children, adolescents, and young adults with acute myeloid leukemia AML who have relapsed once or twice. This phase 3 randomized trial focuses on patients in first relapse who cannot receive more anthracyclines or those in second relapse. The study investigates if targeting the BCL-2 protein with venetoclax can overcome chemotherapy resistance and improve outcomes for this population with poor prognosis. Participants are randomly assigned to two treatment groups. One group receives the chemotherapy combination alone during two 42-day cycles, while the other group receives venetoclax plus the same chemotherapy. Venetoclax is given orally, with dosing schedules varying during the cycles. After the two cycles, participants may undergo stem cell transplantation or receive maintenance therapy with azacitidine alone or combined with venetoclax for up to 24 cycles, depending on the group and clinical benefit. During the trial, participants undergo assessments including survival tracking for up to five years, response rates, relapse incidence, and adverse events. Blood tests measure venetoclax levels at specific time points to understand how the drug behaves in the body. The study also monitors organ function and overall health status. Participants are followed closely to evaluate the effectiveness and safety of the treatments over time.

Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)