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

A Prospective, Multicenter Clinical Study on The Safety and Efficacy of Avapritinib in The Treatment of Relapsed/Refractory Pediatric Core Binding Factor Acute Myeloid Leukemia (CBF-AML) With KIT Mutation

This study is testing if a new drug called avapritinib can help children with a specific type of leukemia that keeps coming back and has a certain mutation, to see if it works better than the treatments they currently have.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: N/A to 18 Years
• Sex: All
• Sponsor: Children's Hospital of Soochow University (Academic / other)
• Drugs / interventions: avapritinib, dasatinib, gilteritinib, chemotherapy, immunotherapy
• Locations: 12 sites (Hefei, Anhui and 11 other locations)
• Trial ID: NCT06316960 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients under 18 years old diagnosed with relapsed or refractory CBF-AML and possessing a KIT mutation.

Not a fit: Patients who have previously received avapritinib or are currently on other targeted therapies for AML may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve survival rates and quality of life for children with this challenging form of leukemia.

How similar studies have performed: Previous studies have shown promising results with avapritinib in similar contexts, indicating potential for success in this trial.

Eligibility criteria

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 this trial is running

Hefei, Anhui and 11 other locations

• 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)
• The First Affiliated Hospital of Zhengzhou University — Zhengzhou, Henan, China (Not_yet_recruiting)
• Third Xiangya Hospital of Central South University — Changsha, Hunan, China (Not_yet_recruiting)
• XiangYa Hospital Central South University — Changsha, Hunan, China (Not_yet_recruiting)
• Children's Hospital of Soochow University — Suzhou, Jiangsu, China (Recruiting)
• Xuzhou Children's Hospital — Xuzhou, Jiangsu, China (Not_yet_recruiting)
• Qilu Hospital of Shandong University — Jinan, Shandong, China (Not_yet_recruiting)
• Children's Hospital Of Fudan University — Shanghai, Shanghai Municipality, China (Not_yet_recruiting)

Study contacts

• Principal investigator: Shaoyan Hu, MD, PhD — Children 's Hospital of Soochow University
• Study coordinator: Shaoyan Hu, MD, PhD

Conditions AML, Childhood; Relapse/Recurrence; Refractory AML; Core Binding Factor Acute Myeloid Leukemia; C-KIT Mutation; Avapritinib; KIT; CBF AML