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

Recruiting now · Phase 1/Phase 2

Conditions studied: Acute Myeloid Leukemia With T(8;21)(Q22;Q22), Acute Myeloid Leukemia With T(16;16)(P13;Q22), KIT Gene Mutation, Avapritinib

In brief

The goal of this clinical trial is to learn if avapritinib combined with standard induction therapy works to treat newly diagnosed adult acute myeloid leukemia (AML) patients with KIT mutations and t(8;21)(q22;q22.1); inv(16)(p13.1q22) or t(16;16)(p13.1;q22). It will also investigate the safety and tolerability of this combination therapy. The main questions it aims to answer are: To determine the maximum tolerated dose (MTD) and/or recommended Phase II dose (RP2D) of avapritinib combined with chemotherapy by Dose-limiting toxicity (DLT). Does this combination therapy improve the rates of minimal residual disease (MRD) negativity and long-term survival outcomes?

Key facts

Study ID: NCT07028073
Run by: The First Affiliated Hospital of Soochow University
People needed: 78
Starts: 2025-05-15
Expected to finish: 2027-12-31
Last updated by the study team: 2025-06-19

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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%

You may not qualify if…

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 is running

The First Affiliated Hospital of Soochow University — Suzhou, Jiangsu, China (enrolling)

Full record on ClinicalTrials.gov

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