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

ConditionsAcute Myeloid LeukemiaNPM1 MutationIDH1 MutationIDH2 Mutation
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorShen yang

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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);

Disqualifiers

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);

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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

Treatment groups

148 Participants
are divided into 2 treatment groups
Group A: Venetoclax plus Azacitidine (VA regimen)Experimental treatment 1 intervention
Group B: "3+7" induction chemotherapyActive comparator 1 intervention

Trial outcomes

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

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)

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shen yang

Lead sponsor

Ruijin Hospital

Sponsor institution

Source ClinicalTrials.gov

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