A Prospective, Multicenter Clinical Study on The Safety and Efficacy of Avapritinib in The Treatment of Relapsed/Refractory Pediatric Core Binding Factor Acute Myeloid Leukemia (CBF-AML) With KIT Mutation
Status: Recruiting
Location: See all (12) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The purpose of this study is to evaluate the efficacy and safety of avapritinib in relapsed or refractory pediatric core binding factor acute myeloid leukemia with KIT mutation.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 18
Healthy Volunteers: f
View:
• Gender unlimited;
• Under 18 years;
• Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).
• Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11;
• KIT mutation;
• Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;
• Relapsed AML: patients who achieved remission after consolidation therapy or transplantation, FISH confirmed that the fusion gene turned positive, or extramedullary leukemia infiltration;
⁃ Participants must have the ability to understand and be willing to participate in this study and must sign an informed consent form.
Locations
Other Locations
China
Third Xiangya Hospital of Central South University
NOT_YET_RECRUITING
Changsha
XiangYa Hospital Central South University
NOT_YET_RECRUITING
Changsha
Guangzhou Women and Children Medical Center
NOT_YET_RECRUITING
Guangzhou
First Affiliated Hospital Of University of Science and Technology of China
NOT_YET_RECRUITING
Hefei
The Second Hospital of Anhui Medical University
NOT_YET_RECRUITING
Hefei
Qilu Hospital of Shandong University
NOT_YET_RECRUITING
Jinan
Kaifeng Children's Hospital
NOT_YET_RECRUITING
Kaifeng
The First Affiliated Hospital of Guangxi Medical University
NOT_YET_RECRUITING
Nanning
Children's Hospital Of Fudan University
NOT_YET_RECRUITING
Shanghai
Children's Hospital of Soochow University
RECRUITING
Suzhou
Xuzhou Children's Hospital
NOT_YET_RECRUITING
Xuzhou
The First Affiliated Hospital of Zhengzhou University
NOT_YET_RECRUITING
Zhengzhou
Contact Information
Primary
Shaoyan Hu, MD, PhD
hsy139@126.com
+86-13771870462
Backup
Li Gao, MD
joygaoli@163.com
+86-15821963190
Time Frame
Start Date:2024-03-01
Estimated Completion Date:2027-03
Participants
Target number of participants:50
Treatments
Experimental: Relapsed/Refractory CBF-AML with KIT mutation
The relapsed/refractory patients will receive a combination treatment of decitabine/azacitidine+ IdAG (idarubicine + cytarabine + granulocyte stimulating factor)regimen along with avapritinib. CBF-AML with KIT mutated patients with molecular relapse after hematopoietic stem cell transplantation may receive avapritinib combined with demethylating agents or interferon or donor lymphocyte infusion without low-dose chemotherapy. The dose of avapritinib will start at 50mg/m2/d, and if platelets stabilize at 50 ×10\^9/L and neutrophils stabilize above 1.0 ×10\^9/L after one week, the dose can be increased to 100mg/m2/d, with a maximum daily dose of 100mg. Avapritinib should be discontinued in the presence of febrile neutropenia or active infection, and avapritinib can be resumed once the infection is controlled, with each treatment cycle not exceeding 28 days.