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.
Can adding quizartinib to standard chemo help adults with a common type of acute myeloid leukemia?
Plain Language Summary
What this means for you:
A trial tested adding quizartinib to standard chemo for a common AML type to see if it improves survival. What this means for you:
A trial tested adding quizartinib to standard chemo for a common AML type to see if it improves survival. View Original Abstract ↓
Status: COMPLETED | Phase: PHASE2
Condition(s): Acute Myeloid Leukemia
Intervention(s): Quizartinib (DRUG), Placebo oral tablet (DRUG), Cytarabine (DRUG), Idarubicin (DRUG)
Randomized phase II trial to compare 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
Detailed: Multicenter, prospective, randomized, placebo-controlled, double-blinded phase II trial to assess the efficacy and safety of an oral quizartinib vs. placebo containing front-line chemotherapy-based schedule in FMS-like tyrosine kinase 3 internal tandem duplications (FLT3-ITD) wild-type Acute Myeloid Leukemia patients.
The trial will be conducted in two phases:
An open-label safety run-in phase: Cytarabine 200 mg/m2 (days 1-7), Idarubicin 12 mg/m2 (days 1-3) Quizartinib 60 mg/d x 14 days (30mg with strong Cytochrome P450 Family 3 Subfamily A (CYP3A) inhibitor) in a total of 9 patients, being observed during 1 cycle of induction to define the final dose for the randomized phase.
A randomized double-blinded phase 2:1 quizartinib (at the established dose) vs. placebo.
Experimental Arm: Cyt
Primary Outcome(s): Event-free survival rate
Enrollment: 273 (ACTUAL)
Lead Sponsor: PETHEMA Foundation
Start: 2019-09-05 | Primary Completion: 2024-10-03