Phase III Study Comparing HMPL-306 to Salvage Chemotherapy in Adults With IDH1- or IDH2-Mutated Relapsed or Refractory Acute Myeloid Leukemia

What this Trial Is About

Researchers are evaluating the drug HMPL-306 compared to salvage chemotherapy in patients with relapsed or refractory acute myeloid leukemia AML who have mutations in the IDH1 or IDH2 genes. This phase III, multicenter, open-label study divides patients into two groups based on their specific IDH mutation and investigates the effectiveness, safety, and pharmacokinetics of HMPL-306. The trial aims to provide new treatment options for AML patients with these genetic mutations who have not responded to prior therapies. Patients are randomly assigned to receive either HMPL-306 monotherapy or one of several salvage chemotherapy regimens selected by their investigator. HMPL-306 is given orally at 250 mg once daily for the first 28-day cycle, then 150 mg once daily for subsequent cycles. Salvage chemotherapy options include intensive regimens like EAMitox and FLAGIda, or less intensive treatments like azacitidine and LoDAC. Treatment continues in 28-day cycles until treatment failure or other study-defined endpoints. Participants will undergo genetic testing to confirm IDH mutation status before enrollment and will be closely monitored throughout the study. This includes bone marrow biopsies, quality of life assessments, and evaluations of treatment response and safety using established criteria. Follow-up extends beyond treatment discontinuation, including safety monitoring and long-term tracking of survival and disease progression. The total study duration may last up to approximately 33 months depending on individual patient outcomes and study progress.

A Study to Evaluate HMPL-306 in Patients With IDH1or IDH2-mutated Acute Myeloid Leukemia