Clinical trial · Interventional
A New Treatment of Newly Diagnosed KIT Mutation CBF-Acute Myeloid Leukemia
Avapritinib Combined With Standard Therapy for the Treatment of Newly Diagnosed KIT Mutation Acute Myeloid Leukemia With t(8;21)(q22;q22.1); Inv(16)(p13.1q22) or t(16;16)(p13.1;q22): a Prospective, Multi-center, Single-arm, Two-cohorts Study
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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?
Conditions
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myeloid Leukemia With T(16;16)(P13;Q22) | Acute Myeloid Leukemia with t(16;16)(p13.1;q22); CBFB-MYH11 | ALIAS | 0.90 |
| Acute Myeloid Leukemia With T(8;21)(Q22;Q22) | Acute Myeloid Leukemia with t(8;21)(q22;q22.1); RUNX1-RUNX1T1 | ALIAS | 0.90 |
| Avapritinib | — | UNRESOLVED | — |
| KIT Gene Mutation | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Group A (FIT): Avapritinib + IA regimen | Drug | — | UNRESOLVED |
| Group B (UNFIT): Avapritinib + VA regimen | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Group A (FIT): Avapritinib Combined Intensive Chemotherapy in patients with newly diagnosed AML
- interventionNames
- Drug: Group A (FIT): Avapritinib + IA regimen
- type
- EXPERIMENTAL
- label
- Group B (UNFIT): Avapritinib Combined Venetoclax/Azacitidine in patients with newly diagnosed AML
- interventionNames
- Drug: Group B (UNFIT): Avapritinib + VA regimen
Primary outcomes (2)
- measure
- Phase I: Recommended phase 2 dose (RP2D)
- timeFrame
- Approximately 6 months after first patient first visit (FPFV)
- description
- Recommended phase 2 dose (RP2D) of IA-based and VA-based combination with avapritinib schedules
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available