Avapritinib with standard chemotherapy for newly diagnosed KIT‑mutant CBF‑AML

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 phase I–II trial enrolls adults with newly diagnosed core‑binding factor AML who have KIT mutations and assigns them to a fit arm (avapritinib plus IA: cytarabine + idarubicin) or an unfit arm (avapritinib plus VA: azacitidine + venetoclax). Phase I uses dose escalation to determine the maximum tolerated dose and recommended phase II dose of avapritinib for each combination, and phase II studies efficacy and safety. Treatment includes induction, consolidation, and maintenance phases with sequential administration designed to manage overlapping toxicities. Key endpoints include dose‑limiting toxicities, MRD negativity, complete remission rates, relapse‑free survival, and overall survival.

Why it matters

Who can join: the full rules

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

Where it's running

• The First Affiliated Hospital of Soochow University
  Suzhou, Jiangsu, China Recruiting

Study contacts

• Study coordinator: Su-ning Chen, M.D.
• Email: chensuning@sina.com
• Phone: 008613814881746

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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.