Randomized Phase 3 Trial Comparing Gilteritinib and Sorafenib as Post-Transplant Maintenance Therapy for FLT3-ITD Positive Acute Myeloid Leukemia Patients Aged 14 to 70

What this Trial Is About

Researchers are evaluating maintenance therapies for patients with FLT3-ITD mutation-positive acute myeloid leukemia AML who have undergone allogeneic hematopoietic stem cell transplantation HCT. This phase 3, open-label, randomized controlled trial compares two drugs, gilteritinib and sorafenib, to determine their effectiveness in preventing disease recurrence. The study includes 594 participants, all tested for minimal residual disease MRD using a central PCR-NGS method to ensure consistent results. Participants are randomly assigned in equal numbers to receive either gilteritinib or sorafenib as maintenance therapy starting between 60 and 90 days after stem cell infusion. Those in the gilteritinib group take three 40 mg tablets daily every morning, while those in the sorafenib group take 400 mg orally twice daily. The treatment phase lasts for two years, with continuous administration of the assigned drug. Throughout the study, participants undergo regular assessments including MRD testing to monitor disease recurrence and other safety evaluations. Researchers will track measurable residual disease recurrence-free survival as the primary outcome over two years, along with several secondary outcomes such as overall survival, relapse rates, and graft-versus-host disease incidence. Follow-up continues until death, withdrawal, or two years after treatment begins, whichever occurs first.

MRD-guided Maintenance Post-HCT: Gilteritini vs Sorafenib