A Phase 2 interventional study of VEN combined with azacitidine and Cytarabine plus Daunorubicin in Acute Myeloid Leukemia, NPM1 Mutation and IDH1 Mutation, sponsored by Shen yang. Not yet recruiting at 1 site in China. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-06-24.
Sponsored by Shen yang · Phase 2, Interventional, and Treatment
This prospective, multicenter, randomized, open-label, non-inferiority clinical study aims to compare the efficacy and safety of VA regimen (venetoclax combined with azacitidine) versus conventional "3+7" chemotherapy regimen in adult patients aged 18 to 65 years with newly diagnosed acute myeloid leukemia (AML) carrying NPM1, IDH1 or IDH2 gene mutations.
The primary goal of this trial is to check whether the VA treatment can reach a non-inferior composite complete remission rate at the end of the induction treatment cycle, which is the key primary endpoint of this research. Several secondary clinical outcomes will also be evaluated in this study, including the rate of minimal residual disease (MRD) negativity after remission, duration of remission, 1-year event-free survival rate and 1-year overall survival rate of enrolled patients. In addition, the safety and treatment-related side effects occurring during the whole induction treatment phase will be systematically collected and compared between two groups as another important secondary assessment.
Eligible enrolled participants will be randomly split into two study groups: patients in experimental group will receive venetoclax plus azacitidine (VA regimen), while patients in control group will receive standard "3+7" induction chemotherapy following conventional clinical protocol. All subjects will complete regular disease assessment, laboratory examinations and scheduled follow-up visits as required by trial design during treatment and post-treatment observation period.
Researchers will collect and analyze all above clinical outcome data from all participants, to verify the non-inferior efficacy and relative safety of VA regimen for this specific subtype of newly diagnosed AML patients.
2,971 studies on the registry are indexed under Leukemia, Myeloid, Acute; 745 are open to participants now.
This study's planned enrollment of 148 is above the median of 41 across 2,509 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →This is the only study on the registry with Shen yang as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Having sufficient organ function, defined as follows:
Exclusion Criteria:
1. Venetoclax: oral administration, specified dosage and schedule for induction cycle; 2. Azacitidine: subcutaneous/intravenous injection with standard induction dose and treatment cycle per clinical protocol.
Drug: VEN combined with azacitidine
Standard 3+7 induction chemotherapy (Cytarabine continuous infusion for 7 days plus Anthracycline intravenous infusion for 3 days) following routine clinical induction regimen for AML.
Drug: Cytarabine plus Daunorubicin
Venetoclax,oral targeted anti-BCL-2 agent and Azacitidine,hypomethylating agent, given via injection for experimental VA arm only
Cytarabine,continuous intravenous infusion for total 7 days and Daunorubicin,Intravenous anthracycline chemotherapy administered for 3 days,in standard 3+7 induction regimen
Composite complete remission rate at the end of induction cycle
Time frame: At the end of 1-2 induction treatment cycles (each cycle is 28 days)
Composite Complete Remission
Time frame: At the end of 1-2 induction treatment cycles (each cycle is 28 days)
Minimal residual disease (MRD) negative rate after remission
Time frame: At the end of induction cycle (each cycle is 28 days)
Duration of Response (DoR)
Time frame: From date of confirmed complete response (CR) until documented disease relapse or death from any cause, assessed up to 24 months after randomization
1-year Event-Free Survival (EFS) rate
Time frame: From date of randomization until first documented treatment failure, relapse, or death from any cause, assessed up to 24 months after randomization; 1-year rate calculated at 12 months post-randomization
1-year Overall Survival (OS) rate
Time frame: From date of randomization until death from any cause, assessed up to 24 months after randomization; 1-year rate calculated at 12 months post-randomization
1-year recurrence free survival rate
Time frame: From date of randomization until first confirmed disease recurrence or death from any cause, assessed up to 24 months after randomization; the 1-year rate will be calculated at the 12-month timepoint after randomization
Safety profile during induction therapy
Evaluate the incidence of grade 3 and grade 4 adverse events classified per CTCAE Version 5.0, the duration of severe adverse events, type, frequency and management of all treatment-emergent adverse events occurring during the induction treatment phase.
Time frame: From initiation of induction therapy through the end of the induction treatment period,assessed up to 8 weeks after randomization
Correlation between baseline AML gene mutation status detected by next-generation sequencing and anti-leukemic efficacy endpoints (complete remission rate, MRD-negative rate, 1-year recurrence-free survival rate) in VA regimen arm
Time frame: From date of randomization up to 24 months after randomization, biomarker-efficacy correlation analysis will be performed using clinical data collected within the first 12 months post randomization
Hospitalization duration during induction therapy
Time frame: From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
Transfusion volume during induction therapy
Time frame: From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
Medical cost during induction therapy
Time frame: From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
Plan to share: No
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This study is not yet recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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