Avapritinib in CBF-AML With KIT Mutations

NCT05821738 | Unknown Phase 2 DMC

• 1 other identifier: SZ-AML-KIT
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Composite complete remission (CRc)

  The proportion of participants who achieve Composite complete remission (CRc)，which includes complete remission (CR)、CR with partial hematologic recovery (CRh)、CR with incomplete blood count recovery (CRi) and morphology leukemia free (MLFS) based on response criteria for AML.

  Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.

Secondary Outcomes (4)

• MRD-negative rate

  Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.

• Progression-free survival (PFS)

  From the first day of treatment until any failure (resistant disease, relapse, or death), assessed up to 1 to 3 years.

• Overall survival (OS)

  From the first day of treatment to time of death from any cause, assessed up 1 to 3 years.

• Incidence of adverse events (AEs)

  Up to approximately 1 to 3 years.

Study Arms (1)

Treatment Group

EXPERIMENTAL

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

Drug: Avapritinib

Interventions

Avapritinib DRUG

administered orally

Treatment Group

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with acute myeloid leukemia accompanied by t(8; 21)/RUNX1-RUNX1T1, or inv(16)/t(16; 16)/CBFβ-MYH11;
• Accompanied by KIT mutation
• Disease recurrence after the first remission, or the mol-MRD remains positive after the morphologic remission of AML.
• No active infection.
• Liver function: TBIL≤ 2×ULN,ALT/AST≤ 3×ULN, CCr ≥ 50ml/min，NYHA grading ≤2; SaO2 \>92%.
• ECOG \<2;
• (11) Predicted survival \> 12 weeks.

You may not qualify if:

• Accept other AML targeted therapies, such as dasatinib, sorafenib, gilteritinib, etc. simultaneously;
• The presence of uncontrolled and active infections (including bacterial, fungal or viral infections).
• Underlying diseases such as myocardial infarction, chronic heart failure, decompensated liver dysfunction, renal failure, etc.
• Pregnant or lactating women;
• Accompanied by other malignant tumors requiring treatment;
• Other interventional clinical studies have been enrolled.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215000, China

RECRUITING

MeSH Terms

Interventions

avapritinib

Study Officials

• Suning Chen

  First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Suning Chen

CONTACT

+8613814881746; chensuning@sina.com

Haiping Dai

CONTACT

13914086271; daihaiping@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: April 7, 2023
• First Posted: April 20, 2023
• Study Start: June 1, 2022
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2025
• Last Updated: May 9, 2023

Record last verified: 2023-05

Locations

CN (1)