Intermediate-dose HAD Regimen for CEBPA Double-mutated AML
NCT Number: NCT06529250
Acronym: HADCEBPA2023
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: NA
Conditions: AML
Interventions: DRUG: HAD|DRUG: Daunorubicin+Cytarabine
Sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sex: ALL
Age: CHILD, ADULT
Enrollment: 148
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-08-13
Completion Date: 2028-09-01
Locations: Blood Hospital, Tianjin, Tianjin, 300020, China

Summary:
AML is highly heterogeneous in pathogenesis, and CEBPA double-mutated (CEBPAdm) AML is a common type of leukemia in China. Currently, no targeted therapies for CEBPAdm, and chemotherapy and transplantation are still the treatment options for CEBPA double-mutated AML. At present, the "3+7" treatment induction regimen of cytarabine combined with anthracyclines is still the first-line recommended regimen. In our retrospective study, the intermediate dose HAD regimen produced a 3-year RFS of 84.7% and a 3-year OS of 92.8% in CEBPAdm AML. Therefore, this project intends to confirm the efficacy of intermediate-dose HAD in the treatment of CEBPA double-mutated AML is superior to the conventional treatment regimen through the multi-center RCT study.

Primary Outcome Measures: Event-free survival (EFS), The interval from randomization to assessment of response after the second course of chemotherapy treatment if patients failed to achieve CR after two courses of induction therapy, the date of relapse, or the date of death, whichever occurred first., up to 2 years after the date of the last enrolled participants
Secondary Outcome Measures: Complete response rate (CR), The proportion that reaches CR status after two courses of induction therapy. Patients should be morphologically free of leukemia, and free of extramedullary leukemia. Absolute neutrophil counts was greater than 1.0\*10\^9/L, and platelet counts was greater than 100\*10\^9/L., Six weeks after induction therapy|30-day mortality, Percentage of patients who died within 30 days from randomization, Within 30 days of randomization|overall survival, The interval from the date of randomization to the date of death or the date of last follow-up for surviving patients., up to 2 years after the date of the last enrolled participants|Event-free survival censored at hematopoietic stem cell transplantation, The interval from randomization to assessment of response after the second course of chemotherapy treatment if patients failed to achieve CR after two courses of induction therapy, the date of relapse, or the date of death, or the date of last follow-up, or the date of hematopoietic stem cell transplantation, whichever occurred first., up to 2 years after the date of the last enrolled participants|Relapse free survival censored at hematopoietic stem cell transplantation, The interval from CR to the date of relapse, or the date of death, or the date of last follow-up, or the date of hematopoietic stem cell transplantation, whichever occurred first. This outcome analyze patients achieved CR in two courses induction therapy., up to 2 years after the date of the last enrolled participants|overall survival censored at hematopoietic stem cell transplantation, It is defined as the time from randomization to the date of death or the date of last follow-up, or the date of hematopoietic stem cell transplantation, whichever occurred first., up to 2 years after the date of the last enrolled participants|60-day mortality, Percentage of patients who died within 60 days from randomization, Within 60 days of randomization|Relapse free survival(RFS), The interval from CR to the date of relapse, or the date of death, or the date of last follow-up, whichever occurred first. This outcome analyze patients achieved CR in two courses induction therapy., up to 2 years after the date of the last enrolled participants