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.

Active, not recruiting

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

Goal

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.

Background

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.

This study in now closed for inclusion.

Last reviewed

March 23, 2026

Study details
Official title
Phase 1/2, multi-center, dose-escalating study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of quizartinib administered in combination with re-induction chemotherapy, and as a single-agent maintenance therapy, in pediatric relapsed/refractory AML subjects aged 1 month to < 18 years (and young adults aged up to 21 years) with FLT-3-ITD mutations
Cancer type
Acute myeloid leukemia(AML) with FLT3-ITD mutations
Phase
1/2
Maximum number of patients
52, of whom 2 are expected to participate in the Netherlands
Start date
January 1, 2019
Status
Closed for inclusion
Local principal investigator
Prof. dr. C.M. Zwaan
Sponsor
Daiichi Sankyo
Approval
The study of this new treatment has been reviewed by an accredited medical research ethics committee. This committee has decided that it is justified to ask patients to participate in this study. More information can be found at: CCMO.
Trial registry number
ClinialTrials.gov: NCT03793478