Avapritinib in CBF-AML With KIT Mutations

NCT ID: NCT05821738

Last Updated: 2023-05-09

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-06-01
• Study Completion Date: 2025-12-31

Brief 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.

Detailed Description

Acute Myeloid Leukemia (AML) with the chromosomal abnormality of t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as the Core Binding Factor AML (CBF-AML). KIT mutation is a common mutation in CBF-AML, which is more likely to relapse and have a worse prognosis.

Avapritinib is an oral tyrosine kinase inhibitor (TKI) with selective inhibitory activity against KIT and PDGFRA. Avapritinib has been approved by FDA for the treatment of gastrointestinal stromal tumors(GIST) with PDGFRA mutations and Advanced systemic mastocytosis (AdvSM). However, the efficacy of avapritinib in AML with KIT mutations is uncertain.

This prospective, multicenter clinical study of the efficacy and safety of avapritinib in relapsed refractory or molecular minimal residual disease (MRD)-positive AML with KIT mutations.

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment Group

The treatment group will receive avapritinib orally. The dosage is 100mg to 300mg qd, allowed to combine with other chemotheray drugs.

Group Type: EXPERIMENTAL

Avapritinib

Intervention Type: DRUG

administered orally

Interventions

Avapritinib

administered orally

Intervention Type: DRUG

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.

• Minimum Eligible Age: 15 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The First Affiliated Hospital of Soochow University

OTHER

Sponsor Role: lead

Responsible Party

Chen Suning

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Suning Chen

Role: PRINCIPAL_INVESTIGATOR

First Affiliated Hospital of Soochow University

Locations

First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, China

Site Status: RECRUITING

Countries

China

Central Contacts

Suning Chen

Role: CONTACT

chensuning@sina.com

+8613814881746

Haiping Dai

Role: CONTACT

daihaiping@126.com

13914086271

Facility Contacts

Suning Chen, PhD

Role: primary

chensuning@sina.com

+8613814881746

Haiping Dai

Role: backup

daihaiping@126.com

13914086271

Other Identifiers

SZ-AML-KIT

Identifier Type: -

Identifier Source: org_study_id