Actively Recruiting
Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric Core Binding Factor Acute Myeloid Leukemia With KIT Mutation
Led by Children's Hospital of Soochow University · Updated on 2024-08-22
50
Participants Needed
12
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of avapritinib in children with relapsed or refractory core binding factor acute myeloid leukemia (CBF-AML) that has a KIT mutation. This phase 2, multicenter, single-arm clinical trial aims to address the challenge that about 30% of CBF-AML patients relapse despite current treatments. KIT mutations, especially the D816V variant, are linked to relapse and poorer survival, and avapritinib has shown promise in related conditions and preliminary reports. Participants will receive a combination treatment including avapritinib alongside hypomethylating agents and low-dose chemotherapy drugs such as decitabine or azacitidine, idarubicin, cytarabine, and granulocyte colony-stimulating factor. The avapritinib dose starts at 50mg/m2 daily and may be increased to 100mg/m2 based on blood counts, with treatment cycles lasting up to 28 days. For patients with molecular relapse after stem cell transplantation, avapritinib may be combined with demethylating agents, interferon, or donor lymphocyte infusion without chemotherapy. During the study, participants will be closely monitored for response to treatment, with the main outcome measured between days 28 and 35 after starting therapy. Researchers will also assess overall survival and progression-free survival for up to five years. Safety will be tracked through clinical evaluations and laboratory tests. The total study duration extends until March 2027, providing long-term follow-up to better understand the treatment's impact.
CONDITIONS
Brief Title
Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Gender unlimited
- Under 18 years old
- Diagnosed with acute myeloid leukemia according to 2022 WHO classification
- Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11
- KIT mutation present
- Refractory AML: no complete remission after induction therapy
- Relapsed AML: remission achieved after consolidation or transplantation with molecular or extramedullary relapse
- No active infections
- Liver function: total bilirubin ≤2 times upper normal limit; ALT/AST ≤3 times upper normal limit; creatinine clearance ≥50 ml/min
- ECOG performance status less than 2
- Expected survival time greater than 12 weeks
- Ability to understand and willing to participate with signed informed consent
You will not qualify if you...
- Prior treatment with avapritinib
- Receiving other targeted AML therapies simultaneously (e.g., dasatinib, sorafenib, gilteritinib, venetoclax)
- Active uncontrolled infections (bacterial, fungal, or viral)
- Significant underlying organ diseases such as myocardial infarction, chronic heart failure, or decompensated liver or kidney dysfunction
- Other malignancies requiring treatment
- Participation in another interventional clinical study
- Determined unsuitable for participation by researchers
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive a combination treatment including avapritinib, hypomethylating agents (decitabine or azacitidine), and low dose chemotherapy (idarubicin, cytarabine, and granulocyte colony-stimulating factor) over repeated 28-day cycles.
Visits occur throughout each 28-day treatment cycle
Duration - Up to 60 months after enrollment
Participants are monitored for overall survival and progression-free survival after completing treatment.
Periodic visits for assessments during follow-up
Trial Site Locations
Total: 12 locations
1
First Affiliated Hospital Of University of Science and Technology of China
Hefei, Anhui, China, 230000
Not Yet Recruiting
2
The Second Hospital of Anhui Medical University
Hefei, Anhui, China, 230000
Not Yet Recruiting
3
Guangzhou Women and Children Medical Center
Guangzhou, Guangdong, China, 510000
Not Yet Recruiting
4
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China, 530000
Not Yet Recruiting
5
Kaifeng Children's Hospital
Kaifeng, Henan, China, 475000
Not Yet Recruiting
6
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, China, 450052
Not Yet Recruiting
7
Third Xiangya Hospital of Central South University
Changsha, Hunan, China, 410000
Not Yet Recruiting
8
XiangYa Hospital Central South University
Changsha, Hunan, China, 410008
Not Yet Recruiting
9
Children's Hospital of Soochow University
Suzhou, Jiangsu, China, 215000
Actively Recruiting
10
Xuzhou Children's Hospital
Xuzhou, Jiangsu, China, 221000
Not Yet Recruiting
11
Qilu Hospital of Shandong University
Jinan, Shandong, China, 250000
Not Yet Recruiting
12
Children's Hospital Of Fudan University
Shanghai, Shanghai Municipality, China, 200000
Not Yet Recruiting
Research Team
S
Shaoyan Hu, MD, PhD
L
Li Gao, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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