Trial Title:
Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation
NCT ID:
NCT06316960
Condition:
AML, Childhood
Relapse/Recurrence
Refractory AML
Core Binding Factor Acute Myeloid Leukemia
C-KIT Mutation
Conditions: Official terms:
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Recurrence
Cytarabine
Azacitidine
Decitabine
Idarubicin
Lenograstim
Conditions: Keywords:
Avapritinib
KIT
CBF AML
Childhood
Relapsed/Refractory
Study type:
Interventional
Study phase:
Phase 2
Overall status:
Recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
Avapritinib
Description:
50mg/m2/day for weighing bodyweight >10kg, 1.65mg/kg/day for weighing ≤ 10kg, po, qd,
d1-28.
Arm group label:
Relapsed/Refractory CBF-AML with KIT mutation
Other name:
AYVAKIT
Intervention type:
Drug
Intervention name:
Azacitidine Injection
Description:
75mg/m2/d for weighing >10kg, 2.5mg/kg/d for weighing ≤ 10kg, d1-7, ivgtt, qd, more than
3 hours. Azacitidine and decitabine cannot be used simultaneously.
Arm group label:
Relapsed/Refractory CBF-AML with KIT mutation
Other name:
Azacitidine
Intervention type:
Drug
Intervention name:
Decitabine Injection
Description:
20mg/m2/d for weighing >10kg, 0.67mg/kg/d for weighing ≤ 10kg, d1-5, ivgtt, qd, more than
3 hours. Azacitidine and decitabine cannot be used simultaneously.
Arm group label:
Relapsed/Refractory CBF-AML with KIT mutation
Other name:
Decitabine
Intervention type:
Drug
Intervention name:
Idarubicin Hydrochloride
Description:
5mg/m2/day for weighing >10kg, 0.17mg/kg/day for weighing ≤ 10kg, d 6, 8, 10 (d 8, 10, 12
for azacitidine) ivgtt, qod, more than 1 hour at 10 am.
Arm group label:
Relapsed/Refractory CBF-AML with KIT mutation
Other name:
Idarubicine
Intervention type:
Drug
Intervention name:
Cytarabine
Description:
10mg/m2/day for weighing >10kg, 0.33mg/kg/day for weighing ≤ 10kg, d6-15 （d8-17 for
azacitidine ）, s.c., q12h.
Arm group label:
Relapsed/Refractory CBF-AML with KIT mutation
Other name:
CYTOSAR
Intervention type:
Drug
Intervention name:
Granulocyte Colony-Stimulating Factor
Description:
300ug/day for weighing >10kg, 10ug/kg/day for weighing ≤10kg, d0-5, s.c., qd.
Arm group label:
Relapsed/Refractory CBF-AML with KIT mutation
Other name:
Recombinant Human Granulocyte Colony-Stimulating Factor Injection
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.
Detailed description:
This is a multicenter, single-arm, prospective, and intervention trial. About 30% of core
binding factor acute myeloid leukemia (CBF-AML) patients still relapse under current
treatment. Some studies have found that KIT mutations, especially the D816V mutation, may
predict relapse and decrease overall survival (OS) in CBF-AML. Avapritinib has been
approved for the treatment of gastrointestinal stromal tumors with KIT or PDGFRA
mutations. Avapritinib was also effective for the treatment of minimal residual disease
in acute myeloid leukemia with t (8;21) and KIT mutation failing to immunotherapy after
allogeneic hematopoietic stem cell transplantation in a single-center, retrospective
report. 11 centers from China carry out the AVACBFKIT regimen including Avapritinib,
hypomethylating agents and low dose chemotherapy for the treatment of relapsed or
refractory pediatric CBF-AML with KIT mutation. The main focus of this study is to
evaluate the efficacy and safety of avapritinib in the regimen.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
1. Gender unlimited;
2. Under 18 years;
3. Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).
4. Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11;
5. KIT mutation;
6. Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;
7. Relapsed AML: patients who achieved remission after consolidation therapy or
transplantation, FISH confirmed that the fusion gene turned positive, or
extramedullary leukemia infiltration;
8. No active infections;
9. Liver function: Tbil ≤2×ULN, ALT/AST ≤3×ULN, creatinine clearance ≥50ml/min;
10. ECOG score <2;
11. Expected survival time >12 weeks;
12. Participants must have the ability to understand and be willing to participate in
this study and must sign an informed consent form.
Exclusion Criteria:
1. Have received prior treatment with avapritinib;
2. Receiving other targeted therapies for AML at the same time, such as dasatinib,
sorafenib, gilteritinib, venetoclax, etc;
3. Presence of active uncontrolled infection (including bacterial, fungal, or viral
infection);
4. Present of significant underlying organ diseases: such as myocardial infarction,
chronic heart failure, decompensated liver or kidney dysfunction；
5. With other malignancies requiring treatment；
6. Already enrolled in another interventional clinical study；
7. The researchers determined that the individual is not suitable to participate in
this trial.
Gender:
All
Minimum age:
N/A
Maximum age:
18 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
First Affiliated Hospital Of University of Science and Technology of China
Address:
City:
Hefei
Zip:
230000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Chun Li, MD,PhD
Phone:
+86-13865992676
Email:
lichun1230@sohu.com
Facility:
Name:
The Second Hospital of Anhui Medical University
Address:
City:
Hefei
Zip:
230000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Ninglin Wang, MD
Phone:
+86-13721113063
Email:
zwnltt@126.com
Facility:
Name:
Guangzhou Women and Children Medical Center
Address:
City:
Guangzhou
Zip:
510000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Hua Jiang, MD, PhD
Phone:
+86-13533330985
Email:
jiang_hua18@sina.cn
Facility:
Name:
The First Affiliated Hospital of Guangxi Medical University
Address:
City:
Nanning
Zip:
530000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Ning Liao, MD
Phone:
+86-13978812808
Email:
ln12808@163.com
Facility:
Name:
Kaifeng Children's Hospital
Address:
City:
Kaifeng
Zip:
475000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Jixia Luo, MD
Phone:
+86-13592146539
Email:
luojixia@163.com
Facility:
Name:
The First Affiliated Hospital of Zhengzhou University
Address:
City:
Zhengzhou
Zip:
450052
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Yufeng Liu, MD,PhD
Phone:
+86-13673666608
Email:
lyf6012@126.com
Facility:
Name:
Third Xiangya Hospital of Central South University
Address:
City:
Changsha
Zip:
410000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Minghua Yang, MD
Phone:
+86-13973135843
Email:
yamahua123@163.com
Facility:
Name:
XiangYa Hospital Central South University
Address:
City:
Changsha
Zip:
410008
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Liangchun Yang, MD,PhD
Phone:
+86-13974927514
Email:
yangliangchung@163.com
Facility:
Name:
Children's Hospital of Soochow University
Address:
City:
Suzhou
Zip:
215000
Country:
China
Status:
Recruiting
Contact:
Last name:
Shaoyan Hu, MD, PhD
Phone:
+86-13771835430
Email:
hsy139@126.com
Contact backup:
Last name:
Li Gao, MD
Phone:
+86-15821963190
Email:
joygaoli@163.com
Investigator:
Last name:
Shaoyan Hu, MD, PhD
Email:
Principal Investigator
Investigator:
Last name:
Peifang Xiao, MD
Email:
Principal Investigator
Investigator:
Last name:
Li Gao, MD
Email:
Sub-Investigator
Investigator:
Last name:
Jun Lu, MD
Email:
Sub-Investigator
Investigator:
Last name:
Jie Li, MD
Email:
Sub-Investigator
Facility:
Name:
Xuzhou Children's Hospital
Address:
City:
Xuzhou
Zip:
221000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Qi An, MD
Phone:
+86-13814422329
Email:
anqi1974@sina.com
Facility:
Name:
Qilu Hospital of Shandong University
Address:
City:
Jinan
Zip:
250000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Xiuli Ju, MD,PhD
Phone:
+86-13869192944
Email:
shellysdcn@hotmail.com
Facility:
Name:
Children's Hospital Of Fudan University
Address:
City:
Shanghai
Zip:
200000
Country:
China
Status:
Not yet recruiting
Contact:
Last name:
Xiaowen Zhai, MD, PhD
Phone:
+86-18017590808
Email:
zhaixiaowendy@163.com
Start date:
March 1, 2024
Completion date:
March 2027
Lead sponsor:
Agency:
Children's Hospital of Soochow University
Agency class:
Other
Source:
Children's Hospital of Soochow University
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06316960
