Avapritinib in CBF-AML With KIT Mutations

• Status: Recruiting
• Trial end date: 2025-12-31
• Target enrollment: 0
• Participant gender: All

Summary

AML with t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as CBF-AML. KIT mutations are common in CBF-AML, which have a worse prognosis.This study is aimed to evaluate the efficacy of Avapritinib, an highly specific inhibitor of the KIT gene, in CBF-AML with KIT mutations.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The First Affiliated Hospital of Soochow University

Criteria

Inclusion Criteria:

1. Patients with acute myeloid leukemia accompanied by t(8; 21)/RUNX1-RUNX1T1, or inv(16)/t(16; 16)/CBFβ-MYH11;

2. Accompanied by KIT mutation

3. Disease recurrence after the first remission, or the mol-MRD remains positive after the morphologic remission of AML.

4. No active infection.

5. Liver function: TBIL≤ 2×ULN,ALT/AST≤ 3×ULN, CCr ≥ 50ml/min，NYHA grading ≤2; SaO2 >92%.

6. ECOG <2;

(11) Predicted survival > 12 weeks.

Exclusion Criteria:

1. Accept other AML targeted therapies, such as dasatinib, sorafenib, gilteritinib, etc. simultaneously;

2. The presence of uncontrolled and active infections (including bacterial, fungal or viral infections).

3. Underlying diseases such as myocardial infarction, chronic heart failure, decompensated liver dysfunction, renal failure, etc.

4. Pregnant or lactating women;

5. Accompanied by other malignant tumors requiring treatment;

6. Other interventional clinical studies have been enrolled.