Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation

NCT06316960 · tracked via the Priya Life Science China tracker

Sponsor

Children's Hospital of Soochow University

Phase

Phase 2

Started

2024-03-01

Last updated

2024-08-22

Condition(s) studied

AML, ChildhoodRelapse/RecurrenceRefractory AMLCore Binding Factor Acute Myeloid LeukemiaC-KIT Mutation

Investigational drug(s) / intervention(s)

Avapritinib →Azacitidine Injection →Decitabine Injection →Idarubicin HydrochlorideCytarabine →Granulocyte Colony-Stimulating Factor →

Avapritinib: 50mg/m2/day for weighing bodyweight \>10kg, 1.65mg/kg/day for weighing ≤ 10kg, po, qd, d1-28.

Azacitidine Injection: 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.

Decitabine Injection: 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.

Idarubicin Hydrochloride: 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.

Cytarabine: 10mg/m2/day for weighing \>10kg, 0.33mg/kg/day for weighing ≤ 10kg, d6-15 （d8-17 for azacitidine ）, s.c., q12h.

Granulocyte Colony-Stimulating Factor: 300ug/day for weighing \>10kg, 10ug/kg/day for weighing ≤10kg, d0-5, s.c., qd.

Study 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

Sex

ALL

Min age

—

Max age

18 Years

Healthy volunteers

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.

Primary outcome measure(s)

Composite remission rate (CRc) — The evaluation time point is day28-day35 from the start of regimen.
Composite remission rate (CRc), including the sum of the number of patients with complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete blood count recovery (CRi), and morphologically leukemia-free (MLFS) as a percentage of the total number of patients who participated in the efficacy analysis.

Trial sites (12)

Facility	City	Region	Status
First Affiliated Hospital Of University of Science and Technology of China	Hefei	Anhui	Not Yet Recruiting
The Second Hospital of Anhui Medical University	Hefei	Anhui	Not Yet Recruiting
Guangzhou Women and Children Medical Center	Guangzhou	Guangdong	Not Yet Recruiting
The First Affiliated Hospital of Guangxi Medical University	Nanning	Guangxi	Not Yet Recruiting
Kaifeng Children's Hospital	Kaifeng	Henan	Not Yet Recruiting
The First Affiliated Hospital of Zhengzhou University	Zhengzhou	Henan	Not Yet Recruiting
Third Xiangya Hospital of Central South University	Changsha	Hunan	Not Yet Recruiting
XiangYa Hospital Central South University	Changsha	Hunan	Not Yet Recruiting
Children's Hospital of Soochow University	Suzhou	Jiangsu	Recruiting
Xuzhou Children's Hospital	Xuzhou	Jiangsu	Not Yet Recruiting
Qilu Hospital of Shandong University	Jinan	Shandong	Not Yet Recruiting
Children's Hospital Of Fudan University	Shanghai	Shanghai Municipality	Not Yet Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06316960 on ClinicalTrials.gov ↗ ← All trials in China