Using Avapritinib for treating specific types of Acute Myeloid Leukemia with KIT mutations

Avapritinib in Relapsed Refractory or MRD-positive CBF-AML With KIT Mutations

This study is testing if a new drug called Avapritinib can help people with a specific type of Acute Myeloid Leukemia that has KIT mutations and hasn't responded well to previous treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 15 Years and up
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (Academic / other)
• Drugs / interventions: dasatinib, gilteritinib, Avapritinib
• Locations: 1 site (Suzhou, Jiangsu)
• Trial ID: NCT05821738 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of Avapritinib, a targeted oral tyrosine kinase inhibitor, in patients with Core Binding Factor Acute Myeloid Leukemia (CBF-AML) who have KIT mutations. The study focuses on individuals who have relapsed or have minimal residual disease after initial treatment. By specifically targeting the KIT mutation, which is associated with a poorer prognosis, the trial aims to determine if Avapritinib can improve outcomes for these patients. The study is multicenter and prospective, ensuring a diverse patient population and robust data collection.

Who should consider this trial

Good fit: Ideal candidates include patients with relapsed CBF-AML characterized by specific chromosomal abnormalities and confirmed KIT mutations.

Not a fit: Patients currently receiving other targeted therapies for AML or those with uncontrolled infections or severe comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve survival rates and quality of life for patients with CBF-AML and KIT mutations.

How similar studies have performed: While Avapritinib has shown success in treating other conditions, its efficacy in CBF-AML with KIT mutations is still being evaluated, making this a novel approach.

Eligibility 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.

Where this trial is running

Suzhou, Jiangsu

• First Affiliated Hospital of Soochow University — Suzhou, Jiangsu, China (Recruiting)

Study contacts

• Principal investigator: Suning Chen — First Affiliated Hospital of Soochow University
• Study coordinator: Suning Chen

Conditions Core Binding Factor Acute Myeloid Leukemia; KIT Mutation-Related Tumors