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Quizartinib (AML)

Clinical study of the safety and efficacy of quizartinib in combination with chemotherapy in children and young adults with AML and FLT3-ITD mutations.
Who can enter
  • Children and young adults with acute myeloid leukemia (AML) and FLT3-ITD-mutations in whom the disease has recurred (relapse) or did not respond to treatment (refractory)
  • Age: from 1 month up to 21 years old


The goal of the first part of this study is to find the right dose of quizartinib for children and young adults in combination with standard chemotherapy. In this part, we determine what dose of quizartinib is high enough to be effective but without serious side effects. In the second part, we will use this dose to test how effective quizartinib is in treating AML with FLT3-ITD.


In a proportion of children with AML, the disease unfortunately recurs or does not respond sufficiently to treatment. The drug quizartinib could help treat the AML of these children.

The drug quizartinib targets the protein FLT3-ITD, a genetic change that occurs in leukemia cells. FLT3-ITD promotes the growth and survival of leukemia cells. Quizartinib is therefore not chemotherapy but a FLT3 protein inhibitor (targeted therapy).

In previous studies, remission occurred in about 50% of adults with AML and FLT3-ITD treated with quizartinib. Many of these patients were able to continue to stem cell transplantation.

Quizartinib was also studied in a very small group of children. That experience led to this study. In this study, we combine the drug quizartinib with standard chemotherapy for relapsed AML.

In order to participate in a study please refer to your/your child’s doctor.
For international patients: please feel welcome to contact our International Office.

Last reviewed

September 6, 2022