Using Avapritinib to treat relapsed or refractory pediatric CBF-AML with KIT mutation

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety and efficacy of avapritinib in treating pediatric patients with relapsed or refractory core binding factor acute myeloid leukemia (CBF-AML) who have a KIT mutation. The study is a multicenter, single-arm, prospective intervention involving 11 centers in China. It aims to address the high relapse rate in CBF-AML patients by incorporating avapritinib alongside hypomethylating agents and low-dose chemotherapy. The trial will assess how well this regimen works in improving patient outcomes compared to existing treatments.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Gender unlimited;
2. Under 18 years;
3. Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).
4. Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11;
5. KIT mutation;
6. Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;
7. Relapsed AML: patients who achieved remission after consolidation therapy or transplantation, FISH confirmed that the fusion gene turned positive, or extramedullary leukemia infiltration;
8. No active infections;
9. Liver function: Tbil ≤2×ULN, ALT/AST ≤3×ULN, creatinine clearance ≥50ml/min;
10. ECOG score \<2;
11. Expected survival time \>12 weeks;
12. Participants must have the ability to understand and be willing to participate in this study and must sign an informed consent form.

Exclusion Criteria:

1. Have received prior treatment with avapritinib;
2. Receiving other targeted therapies for AML at the same time, such as dasatinib, sorafenib, gilteritinib, venetoclax, etc;
3. Presence of active uncontrolled infection (including bacterial, fungal, or viral infection);
4. Present of significant underlying organ diseases: such as myocardial infarction, chronic heart failure, decompensated liver or kidney dysfunction；
5. With other malignancies requiring treatment；
6. Already enrolled in another interventional clinical study；
7. The researchers determined that the individual is not suitable to participate in this trial.

Where it's running

• Children's Hospital of Soochow University
  Suzhou, Jiangsu, China Recruiting
• First Affiliated Hospital Of University of Science and Technology of China
  Hefei, Anhui, China Not yet recruiting
• The Second Hospital of Anhui Medical University
  Hefei, Anhui, China Not yet recruiting
• Guangzhou Women and Children Medical Center
  Guangzhou, Guangdong, China Not yet recruiting
• The First Affiliated Hospital of Guangxi Medical University
  Nanning, Guangxi, China Not yet recruiting
• Kaifeng Children's Hospital
  Kaifeng, Henan, China Not yet recruiting

Study contacts

• Principal investigator: Shaoyan Hu, MD, PhD — Children 's Hospital of Soochow University
• Study coordinator: Shaoyan Hu, MD, PhD
• Email: hsy139@126.com
• Phone: +86-13771870462

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.