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NCT Number: NCT07664839

Study on the Efficacy and Safety of VA Regimen Compared to "3+7" Regimen in Newly Diagnosed AML With NPM1 or IDH1/IDH2 Mutations

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.

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Key information

Conditions

Acute Myeloid Leukemia Hematologic Diseases Hemic and Lymphatic Diseases IDH1 Mutation IDH2 Mutation Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute NPM1 Mutation Neoplasms Neoplasms by Histologic Type

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200025, China

Location contact

Yang Shen, MD

CONTACT

[email protected]

+86-021-64370045

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 65 years old ≥ 18 years old;
  • Diagnosed as acute myeloid leukemia (non APL) (diagnostic criteria refer to the 2022 ELN classification system);
  • Initial diagnosis accompanied by NPM1 mutations (A, B, D types and rare types are all acceptable) and/or IDH1/IDH2 mutations;
  • Have not received any other induction therapy before (except hydroxyurea);
  • Physical fitness status score (ECOG PS) 0-3;
  • Having sufficient organ function, defined as follows:
  • Liver function: serum total bilirubin ≤ 3 x upper limit of normal range (ULN), aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤ 3 x ULN, unless considered to be caused by leukemia;
  • Renal function: endogenous creatinine clearance rate ≥ 30ml/min;
  • Heart function: NYHA classification ≤ 2 points;
  • Participants must have the ability to understand and be willing to participate in this study, and sign an informed consent form.

Exclusion criteria

  • Acute promyelocytic leukemia;
  • Merge extramedullary infiltration such as central nervous system leukemia;
  • Have a clear history of CMML or MDS, and later progress to AML; Or have a history of malignant tumors;
  • There is uncontrolled active infection (including bacterial, fungal, or viral infections);
  • Pregnant or lactating women;
  • Researchers determine that participants are not suitable to participate in this experiment

Treatment and study plan

VEN combined with azacitidine

Drug

Venetoclax,oral targeted anti-BCL-2 agent and Azacitidine,hypomethylating agent, given via injection for experimental VA arm only

Cytarabine plus Daunorubicin

Drug

Cytarabine,continuous intravenous infusion for total 7 days and Daunorubicin,Intravenous anthracycline chemotherapy administered for 3 days,in standard 3+7 induction regimen

Primary outcomes

  1. 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)

Secondary outcomes

  1. Composite Complete Remission

    Time frame: At the end of 1-2 induction treatment cycles (each cycle is 28 days)

  2. Minimal residual disease (MRD) negative rate after remission

    Time frame: At the end of induction cycle (each cycle is 28 days)

  3. 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

  4. 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

  5. 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

  6. 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

  7. Safety profile during induction therapy

    Time frame: From initiation of induction therapy through the end of the induction treatment period,assessed up to 8 weeks after randomization

    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.

Other outcomes

  1. 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

  2. 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)

  3. 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)

  4. 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)

Study contacts

Contact information is provided by the study sponsor or research team.

Yang Shen, MD

CONTACT

[email protected]

+86-021-64370045

Sponsors and collaborators

Lead sponsor

Shen yang

Other

Registry information

Official study title

A Prospective, Multicenter, Randomized Controlled, Open Label, Non-Inferiority Study Comparing the Efficacy and Safety of the VA Regimen (Venetoclax Combined With Azacitidine) With the "3+7" Regimen in the Treatment of Newly Diagnosed AML Patients With NPM1 or IDH1/IDH2 Mutations

Important dates

Study start
2026
Primary completion
2028
Study completion
2032
First posted
Jun 24, 2026
Registry last updated
Jun 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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