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

Plain-language summary

This study evaluates the combination of Avapritinib with chemotherapy regimens (IA or VA) in patients with newly diagnosed KIT-mutant CBF-acute myeloid leukemia (core binding factor acute myeloid leukemia). It is open to previously untreated patients aged 18 and older who carry certain genetic changes. The study is being conducted in Phase 1/Phase 2 and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

The First Affiliated Hospital of Soochow University

Intervention

Group A (FIT): Avapritinib + IA regimen, Group B (UNFIT): Avapritinib + VA regimen

Start date

5/15/2025

Estimated completion

12/31/2027

Countries

China

Related genes

Locations

• Suzhou, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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