Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 18
SponsorChildren's Hospital of Soochow University

About this trial

The purpose of this study is to evaluate the efficacy and safety of avapritinib in relapsed or refractory pediatric core binding factor acute myeloid leukemia with KIT mutation.

Eligibility criteria

Qualifiers

Gender unlimited;

Under 18 years;

Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).

Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11;

Disqualifiers

Have received prior treatment with avapritinib;

Receiving other targeted therapies for AML at the same time, such as dasatinib, sorafenib, gilteritinib, venetoclax, etc;

Presence of active uncontrolled infection (including bacterial, fungal, or viral infection);

Present of significant underlying organ diseases: such as myocardial infarction, chronic heart failure, decompensated liver or kidney dysfunction;

Trial design

Treatments tested in this trial

  • Avapritinib
  • Azacitidine Injection
  • Decitabine Injection
  • Idarubicin Hydrochloride
  • Cytarabine
  • Granulocyte Colony-Stimulating Factor

Treatment groups

50 Participants
are divided into 1 treatment group