Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mu
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.
| Condition(s) | AML, Childhood, Relapse/Recurrence, Refractory AML, Core Binding Factor Acute Myeloid Leukemia, C-KIT Mutation |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| 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. |
| Who can participate | 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 conse |
| Ages | 18 Years |
| Sex | All |
| Lead sponsor | Children's Hospital of Soochow University |
| Locations | Hefei, Anhui, China; Hefei, Anhui, China; Guangzhou, Guangdong, China; Nanning, Guangxi, China; Kaifeng, Henan, China; Zhengzhou, Henan, China (+6 more sites) |
| Start date | 2024-03-01 |
| NCT ID | NCT06316960 |
| Official listing | https://clinicaltrials.gov/study/NCT06316960 |