Phase 3 Study of Venetoclax with Chemotherapy for Adults Newly Diagnosed with Acute Myeloid Leukemia or Myelodysplastic Syndrome with Excess Blasts-2

What this Trial Is About

This research aims to evaluate the addition of venetoclax to standard induction and consolidation chemotherapy in adults newly diagnosed with acute myeloid leukemia AML or myelodysplastic syndrome with excess blasts-2 MDS-EB-2. It is a randomized, placebo-controlled, double-blind phase 3 clinical trial preceded by a dose-escalation feasibility phase to establish the venetoclax dose. The study is conducted across multiple centers and sponsored by the University of Ulm. Participants first undergo a feasibility run-in phase where venetoclax dosing is determined. Then, eligible patients are randomly assigned to receive either intensive chemotherapy combined with venetoclax or with a placebo. Treatment includes two cycles of induction chemotherapy followed by consolidation therapy based on initial randomization and established protocols. Patients achieving complete remission or certain other response states continue consolidation, and some may undergo allogeneic stem cell transplantation or other treatments depending on clinical factors. During the study, participants receive detailed evaluations including assessments of treatment safety and effectiveness. Researchers monitor event-free survival, dose-limiting toxicities, remission rates, relapse, survival, and quality of life using questionnaires and laboratory testing. Treatment adherence and safety are closely tracked. The study follows participants until at least 16 months after the last patient is enrolled, with some outcome measures extending to 28 months, to gather comprehensive data on treatment impact and patient health.

Phase III Study of Induction and Consolidation Chemotherapy With Venetoclax in Patients With Newly Diagnosed AML or MDS-EB-2