A New Treatment of Newly Diagnosed KIT Mutation CBF-Acute Myeloid Leukemia

Launched by THE FIRST AFFILIATED HOSPITAL OF SOOCHOW UNIVERSITY · Jun 11, 2025

Keywords

ClinConnect Summary

This clinical trial is testing a new treatment for adults who have just been diagnosed with a specific type of acute myeloid leukemia (AML), a blood cancer. The study is looking at whether adding a drug called avapritinib to the usual chemotherapy can help improve treatment results for patients whose cancer cells have certain genetic changes called KIT mutations and specific chromosome changes. The researchers want to find the safest and most effective dose of avapritinib when combined with standard chemotherapy, and see if this combination can better reduce leftover cancer cells and improve long-term survival.

Adults aged 18 and older who are newly diagnosed with this type of AML and meet certain health criteria may be eligible to join. Participants should not have had prior treatment for their leukemia (except for very limited use of some medicines) and must have specific genetic features in their cancer confirmed by testing. The trial is open to people with good overall health as well as those with some other health issues, as long as organ functions like liver, kidney, and heart are still working well enough. During the trial, participants will receive avapritinib along with standard chemotherapy and will be closely monitored for side effects and how well the treatment works. It’s important to note that people who are pregnant, breastfeeding, or have certain serious health problems won’t be able to join.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Age ≥18 years, both genders
• • Diagnosis of acute myeloid leukemia according to WHO 2022 criteria
• • Treatment-naive patients (hydroxyurea or low-dose cytarabine \<0.5g cumulative dose allowed)
• • Bone marrow detection of KIT mutations with concurrent t(8;21)(q22;q22.1) or RUNX1::RUNX1T1 fusion gene; or inv(16)(p13.1q22) or t(16;16)(p13.1;q22) or CBFβ::MYH11 fusion gene
• • Life expectancy \>12 weeks Group A: ≥18 and \<65 years with ECOG 0-1; Group B: ≥65 years or ≥18 and \<65 years with comorbidities (ECOG ≥2, cardiac disease, creatinine clearance 30-50ml/min, or mild hepatic impairment)
• • Adequate organ function: bilirubin ≤2×ULN, ALT/AST ≤3×ULN (≤5×ULN if leukemic infiltration), creatinine clearance ≥30ml/min, left ventricular ejection fraction \>45%
• Exclusion Criteria:
• • Known hypersensitivity to KIT inhibitors, cytarabine, idarubicin, venetoclax, azacitidine or similar agents
• • Concurrent use of other KIT inhibitors (dasatinib, sorafenib, gilteritinib, midostaurin)
• • Intracranial hemorrhage on imaging or unresolved prior intracranial bleeding
• • Active uncontrolled infection
• • Significant organ dysfunction: myocardial infarction, chronic heart failure, decompensated liver dysfunction, renal failure
• • Pregnancy or breastfeeding