Phase II trial compares quizartinib vs placebo with chemotherapy in FLT3 wild-type AML

This multicenter, prospective, randomized, placebo-controlled, double-blinded phase II trial compared the efficacy and safety of standard chemotherapy plus quizartinib versus standard chemotherapy plus placebo in adult patients with newly diagnosed FLT3 wild-type acute myeloid leukemia. The trial enrolled 273 patients and was conducted in two phases. First, an open-label safety run-in phase involved 9 patients receiving cytarabine 200 mg/m² (days 1-7), idarubicin 12 mg/m² (days 1-3), and quizartinib 60 mg/day for 14 days (or 30 mg with a strong CYP3A inhibitor) during one induction cycle to establish the final dose for the randomized phase. Second, a randomized double-blinded phase assigned patients in a 2:1 ratio to receive quizartinib at the established dose versus placebo, both combined with the chemotherapy backbone. The primary outcome measure was event-free survival rate. The study started on September 5, 2019, and reached primary completion on October 3, 2024. The abstract does not report specific efficacy results, safety data, or statistical comparisons between the treatment arms.

For adults newly diagnosed with a specific type of acute myeloid leukemia (AML) called FLT3 wild-type, the standard first treatment is a combination of chemotherapy drugs. This trial asked a straightforward question: what happens if we add another drug, called quizartinib, to that standard mix? The goal was to see if this combination could help patients live longer without their cancer coming back or other major health events—what doctors call 'event-free survival.' The study involved 273 patients and was designed in two parts. First, a small group of 9 patients received the full drug combination to check for safety and determine the best dose of quizartinib to use. Then, the main part of the trial randomly assigned patients to receive either the standard chemotherapy plus quizartinib or the standard chemotherapy plus a placebo pill, with neither the patients nor their doctors knowing which one they got. The trial was completed to compare how well the two approaches worked and to understand their safety profiles. The core finding focuses on whether adding quizartinib made a meaningful difference in event-free survival for these patients.