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

Recruiting now · Phase 2

Conditions studied: AML, Childhood, Relapse/Recurrence, Refractory AML, Core Binding Factor Acute Myeloid Leukemia, C-KIT Mutation

In brief

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.

Key facts

Study ID

NCT06316960

Run by

Children's Hospital of Soochow University

People needed

50

Starts

2024-03-01

Expected to finish

2027-03-01

Last updated by the study team

2024-08-22

Who can join

Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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;
• KIT mutation;
• Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;
• Relapsed AML: patients who achieved remission after consolidation therapy or transplantation, FISH confirmed that the fusion gene turned positive, or extramedullary leukemia infiltration;
• No active infections;
• Liver function: Tbil ≤2×ULN, ALT/AST ≤3×ULN, creatinine clearance ≥50ml/min;
• ECOG score <2;
• Expected survival time >12 weeks;
• Participants must have the ability to understand and be willing to participate in this study and must sign an informed consent form.

You may not qualify if…

• 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；
• With other malignancies requiring treatment；
• Already enrolled in another interventional clinical study；
• The researchers determined that the individual is not suitable to participate in this trial.

Where it is running

• Children's Hospital of Soochow University — Suzhou, Jiangsu, China (enrolling)
• The Second Hospital of Anhui Medical University — Hefei, Anhui, China
• Guangzhou Women and Children Medical Center — Guangzhou, Guangdong, China
• The First Affiliated Hospital of Guangxi Medical University — Nanning, Guangxi, China
• Kaifeng Children's Hospital — Kaifeng, Henan, China
• The First Affiliated Hospital of Zhengzhou University — Zhengzhou, Henan, China
• Third Xiangya Hospital of Central South University — Changsha, Hunan, China
• XiangYa Hospital Central South University — Changsha, Hunan, China
• Xuzhou Children's Hospital — Xuzhou, Jiangsu, China
• Qilu Hospital of Shandong University — Jinan, Shandong, China
• First Affiliated Hospital Of University of Science and Technology of China — Hefei, Anhui, China
• Children's Hospital Of Fudan University — Shanghai, Shanghai Municipality, China

Full record on ClinicalTrials.gov

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