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

Phase 2

Recruiting

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

• Registration Number: NCT06316960
• Lead Sponsor: Children's Hospital of Soochow University

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-3-12
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Inclusion Criteria:<br><br> 1. Gender unlimited;<br><br> 2. Under 18 years;<br><br> 3. Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).<br><br> 4. Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFß::MYH11;<br><br> 5. KIT mutation;<br><br> 6. Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;<br><br> 7. Relapsed AML: patients who achieved remission after consolidation therapy or<br> transplantation, FISH confirmed that the fusion gene turned positive, or<br> extramedullary leukemia infiltration;<br><br> 8. No active infections;<br><br> 9. Liver function: Tbil =2×ULN, ALT/AST =3×ULN, creatinine clearance =50ml/min;<br><br> 10. ECOG score <2;<br><br> 11. Expected survival time >12 weeks;<br><br> 12. Participants must have the ability to understand and be willing to participate in<br> this study and must sign an informed consent form.<br><br>Exclusion Criteria:<br><br> 1. Have received prior treatment with avapritinib;<br><br> 2. Receiving other targeted therapies for AML at the same time, such as dasatinib,<br> sorafenib, gilteritinib, venetoclax, etc;<br><br> 3. Presence of active uncontrolled infection (including bacterial, fungal, or viral<br> infection);<br><br> 4. Present of significant underlying organ diseases: such as myocardial infarction,<br> chronic heart failure, decompensated liver or kidney dysfunction;<br><br> 5. With other malignancies requiring treatment;<br><br> 6. Already enrolled in another interventional clinical study;<br><br> 7. The researchers determined that the individual is not suitable to participate in<br> this trial.

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Composite remission rate (CRc)

Secondary Outcome Measures

Name	Time	Method
Overall survival;Progression-free survival