Acute Myeloid LeukemiaPhase 2NCT07664839
Study on the Efficacy and Safety of VA Regimen Compared to "3+7" Regimen in Newly Diagnosed AML With NPM1 or IDH1/IDH2 Mutations
A Phase 2 study testing VEN combined with azacitidine and Cytarabine plus Daunorubicin in people with Acute Myeloid Leukemia and NPM1 Mutation, run by Shen yang at 1 site in China.
Trial summary
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)… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Acute Myeloid Leukemia, NPM1 Mutation, IDH1 Mutation
- What is being tested?
- VEN combined with azacitidine and Cytarabine plus Daunorubicin
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 148 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2026to 31 May 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200025
Yang Shen, MD, +86-021-64370045, sy_clinicaltrial@163.com
About this study
Official 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
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.
Read more
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.
- Conditions
- Acute Myeloid Leukemia, NPM1 Mutation, IDH1 Mutation, IDH2 Mutation
- Being tested
- VEN combined with azacitidine, Cytarabine plus Daunorubicin
- Keywords
- Acute myeloid leukemia, Venetoclax, NPM1 mutation, IDH1 mutation, IDH2 mutation
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VEN combined with azacitidineDrug
Venetoclax,oral targeted anti-BCL-2 agent and Azacitidine,hypomethylating agent, given via injection for experimental VA arm only
Cytarabine plus DaunorubicinDrug
Cytarabine,continuous intravenous infusion for total 7 days and Daunorubicin,Intravenous anthracycline chemotherapy administered for 3 days,in standard 3+7 induction regimen
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax plus Azacitidine (VA regimen) | Experimental | 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. Receives: Drug: VEN combined with azacitidine |
| "3+7" induction chemotherapy | Active comparator | 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. Receives: Drug: Cytarabine plus Daunorubicin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite complete remission rate at the end of induction cycle
Measured: At the end of 1-2 induction treatment cycles (each cycle is 28 days)
Secondary outcomes (7)
Composite Complete Remission
Measured: At the end of 1-2 induction treatment cycles (each cycle is 28 days)
Minimal residual disease (MRD) negative rate after remission
Measured: At the end of induction cycle (each cycle is 28 days)
Duration of Response (DoR)
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: From initiation of induction therapy through the end of the induction treatment period,assessed up to 8 weeks after randomization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2026
- Study start1 Jul 2026 (estimated)
- Primary completion31 May 2028 (estimated)
- Study completion31 May 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 148 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shen yang
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Shen yang, Ruijin Hospital
- Investigators
- Yang Shen, MD, Principal investigator, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07664839
- Sponsor's protocol ID
- NPM1/IDH-AML01
- Status last verified
- Jun 2026
- First submitted
- 10 Jun 2026
- Last updated
- 24 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07664839
- Acronym / sponsor ID
- NPM1/IDH-AML01
- Conditions
- Acute Myeloid Leukemia, NPM1 Mutation, IDH1 Mutation, IDH2 Mutation
- Keywords
- Acute myeloid leukemia, Venetoclax, NPM1 mutation, IDH1 mutation, IDH2 mutation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 148 participants (estimated)
- Lead sponsor
- Shen yangUniversity, hospital or other
- Study start
- 1 Jul 2026 (estimated)
- Primary completion
- 31 May 2028 (estimated)
- Study completion
- 31 May 2032 (estimated)
- First posted
- 24 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Venetoclax plus Azacitidine (VA regimen) | Experimental | Drug: VEN combined with azacitidine1. 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. |
| "3+7" induction chemotherapy | Active comparator | Drug: Cytarabine plus DaunorubicinStandard 3+7 induction chemotherapy (Cytarabine continuous infusion for 7 days plus Anthracycline intravenous infusion for 3 days) following routine clinical induction regimen for AML. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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;
Key exclusion criteria (6 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VEN combined with azacitidineVenetoclax,oral targeted anti-BCL-2 agent and Azacitidine,hypomethylating agent, given via injection for experimental VA arm only | Drug | Venetoclax plus Azacitidine (VA regimen) |
| Cytarabine plus DaunorubicinCytarabine,continuous intravenous infusion for total 7 days and Daunorubicin,Intravenous anthracycline chemotherapy administered for 3 days,in standard 3+7 induction regimen | Drug | "3+7" induction chemotherapy |
Primary outcome
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)
7 secondary outcomes
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
Time frame: From initiation of induction therapy through the end of the induction treatment period,assessed up to 8 weeks after randomization
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Yang Shen, MD principal investigator
Ruijin Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VEN combined with azacitidine and Cytarabine plus Daunorubicin in people with Acute Myeloid Leukemia and NPM1 Mutation, run by Shen yang at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 24 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yang Shen, MD (+86-021-64370045, sy_clinicaltrial@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2026.