Acute Myeloid LeukemiaPhase 3NCT07407660
Shortened Venetoclax Duration Based on Day 14 BM Blasts Versus Standard Therapy in Elderly or Frail Patients With AML Patients Treated With Azacitidine Plus Venetoclax
A Phase 3 study testing Venetoclax and Azacitidine (AZA) in people with Acute Myeloid Leukemia, run by Peking University People's Hospital.
Trial summary
This study aims to compare the efficacy and safety of a shortened treatment course based on the bone marrow blast count on Day 14 versus standard treatment in patients with acute myeloid leukemia treated with venetoclax plus azacitidine. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Venetoclax and Azacitidine (AZA)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 250 participants
- Where
- No sites listed yet
- Study dates
- 1 Feb 2026to 1 Feb 2030
- Phase
- Phase 3
- 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 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with newly diagnosed acute myeloid leukemia who meet the WHO 2022 criteria.
- Meeting one of the following conditions:
- Aged ≥ 60 years;
- aged ≥ 18 years and < 60 years, with one or more of the following comorbidities that render the subject unsuitable for intensive induction therapy:
- Complicated with congestive heart failure, or left ventricular ejection fraction ≤ 50%, or a history of chronic stable angina pectoris;
- A history of pulmonary disease, with carbon monoxide diffusing capacity of the lung (DLCO) ≤ 65% or forced expiratory volume in 1 second (FEV1) ≤ 65%;
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- Creatinine clearance rate < 45 mL/min (calculated by the Cockcroft-Gault formula);
- Total bilirubin > 1.5 × upper limit of normal;
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) score ≥ 2;
- Any other comorbidities judged by the investigator to contraindicate intensive induction therapy.
- Received induction therapy with the azacitidine plus venetoclax regimen (azacitidine for injection: 75 mg/m² subcutaneously on Days 1-7; venetoclax tablets: 100 mg on Day 1, 200 mg on Day 2, and 400 mg once daily starting on Day 3) for 12-14 days. Dose adjustment of venetoclax shall be performed if combined with strong or moderate CYP3A/P-gp inhibitors.
- Completed risk stratification assessment per the ELN 2022 criteria.
- Signed the informed consent form.
Who cannot join
- Diagnosis of acute promyelocytic leukemia, AML with t(8;21)(q22;q22.1)/ RUNX1::RUNX1T1 translocation, or blast crisis of chronic myeloid leukemia (CML).
- Prior treatment with venetoclax before the diagnosis of acute myeloid leukemia.
- A history of allogeneic hematopoietic stem cell transplantation.
- Severe hepatic or renal impairment, defined by the presence of any of the following abnormalities: aspartate aminotransferase (AST) > 2.5 × ULN; alanine aminotransferase (ALT) > 2.5 × ULN; creatinine clearance rate < 30 mL/min (calculated by the Cockcroft-Gault formula); or total bilirubin > 3 × ULN.
- Presence of acute active infection requiring intravenous systemic therapy.
- Presence of active malignant tumors requiring antineoplastic treatment.
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- Presence of active autoimmune diseases requiring treatment with prednisone ≥ 15 mg/day or equivalent doses of other glucocorticoids, or any other immunosuppressive agents.
- Inability to swallow tablets, or presence of diseases significantly impairing gastrointestinal function (e.g., malabsorption syndrome, gastrectomy or enterectomy, bariatric surgery, symptomatic inflammatory bowel disease, or partial/complete intestinal obstruction).
- Pregnant or lactating female subjects.
- Subjects judged by the investigator to be unable to comply with the protocol due to uncontrollable medical, psychological, familial, social, or geographic conditions; or those who are unwilling or unable to follow the required procedures of the protocol.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Shortened Venetoclax Duration Based on Bone Marrow Blasts on Day 14 Versus Standard Therapy in Elderly or Frail Patients With Acute Myeloid Leukemia Treated With Azacitidine Plus Venetoclax: A Multicenter Prospective Randomized Controlled Study
This study aims to compare the efficacy and safety of a shortened treatment course based on the bone marrow blast count on Day 14 versus standard treatment in patients with acute myeloid leukemia treated with venetoclax plus azacitidine.
Read the detailed description
The standard 28-day cycle of venetoclax is widely recommended for the first cycle of venetoclax plus azacitidine induction therapy in patients with acute myeloid leukemia (AML). However, it has been found that the 28-day treatment cycle was not tolerant for some patients due to severe myelosuppression and infection, which may lead to treatment interruption and delays in subsequent treatment cycles.
This is a multicenter, randomized controlled, open-label, non-inferiority study, which compare the efficacy and safety of a shortened treatment course based on the bone marrow blast count on Day 14 versus standard treatment in AML patients treated with venetoclax plus azacitidine induction therapy.
This study plans to enroll 250 newly diagnosed AML patients who are intolerant to intensive chemotherapy regimens. Enrolled subjects will be assigned to either the optimized treatment group or the standard treatment group in a 1:1 ratio with stratified blocked randomization, with ELN 2022 classification as the stratification factor. In the optimized treatment group, if the bone marrow blast count is <5% on Day 14 of the first induction, the duration of venetoclax will be shortened to 14 days; otherwise, the 28-day course will be completed as scheduled. In the standard treatment group, no bone marrow assessment will be performed on Day 14, and all patients will complete the 28-day treatment course. The duration of venetoclax in the second cycle will be 28 days or 21 days (if complete remission with incomplete hematologic recovery) for the two groups. The primary endpoint is the achievement of complete remission or complete remission with incomplete hematologic recovery (CR/CRi) within 2 treatment courses.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Venetoclax, Azacitidine (AZA)
- Keywords
- Venetoclax, Azacitidine, Early assessment, Treatment optimization, Acute myeloid leukemia
- 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.
VenetoclaxDrug
Tablet
Azacitidine (AZA)Drug
Solution for subcutaneous
| Study group | Role | What they receive |
|---|---|---|
| Optimized treatment | Experimental | Bone marrow aspiration is perfomed on day 14 of the first induction cycle. If the bone marrow blast count is <5%, the duration of venetoclax will be shortened to 14 days; otherwise, the 28-day course will be completed.
In the second cycle, patients who fail to achieve CR/CRi and those who achieve CR will receive a 28-day course of venetoclax plus azacitidine; for patients who achieve CRi, a 21-day course of venetoclax plus azacitidine will be administered within 14 days following the first cycle.
Treatment for subsequent cycles will be determined according to the investigators' local clinical practice, including but not limited to continued venetoclax-azacitidine therapy, switching to intensive chemotherapy, or allogeneic hematopoietic stem cell transplantation. Receives: Drug: Venetoclax; Drug: Azacitidine (AZA) |
| Standard treatment | Active comparator | No bone marrow assessment was performed on day 14 of the first induction cycle. All the patients receive the 28-day course of venetoclax plus azacitidine for the first cycle.
In the second cycle, patients who fail to achieve CR/CRi and those who achieve CR will receive a 28-day course of venetoclax plus azacitidine; for patients who achieve CRi, a 21-day course of venetoclax plus azacitidine will be administered within 14 days following the first cycle.
Treatment for subsequent cycles will be determined according to the investigators' local clinical practice, including but not limited to continued venetoclax-azacitidine therapy, switching to intensive chemotherapy, or allogeneic hematopoietic stem cell transplantation. Receives: Drug: Venetoclax; Drug: Azacitidine (AZA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Achievement of CR/CRi within 2 treatment cycles
Measured: At the end of Cycle 1 and Cycle 2 (each cycle is 28 days). If CRi, repeat 2 weeks later.
Secondary outcomes (6)
Achievement of CR within 2 treatment cycles
Measured: At the end of Cycle 1 and Cycle 2 (each cycle is 28 days). If CRi, repeat 2 weeks later.
Achievement of CR/CRi during treatment with the venetoclax plus azacitidine regimen
Measured: At the end of Cycles 1, 2, 3, and 5 (each cycle is 28 days) of the venetoclax-azacitidine regimen, and every 2 cycles thereafter.
Achievement of MRD negativity during treatment with the venetoclax plus azacitidine regimen
Flow cytometry analysis of bone marrow specimen.
Measured: At the end of Cycles 1, 2, 3, and 5 (each cycle is 28 days) of the venetoclax-azacitidine regimen, and every 2 cycles thereafter.
Relapse-free survival
Relapse-free survival is defined as the number of months from the first achievement of CR/CRi to disease relapse or death from any cause, whichever came first, or censored at the last follow-up.
Measured: From the first achievement of CR/CRi to disease relapse or death from any cause, whichever came first, assessed up to 48 months.
Overall survival
Overall survival is defined as the number of months from enrollment to death from any cause, or censored at the last follow-up.
Measured: Time from enrollment to death from any cause, assessed up to 48 months.
Adverse events
Measured: Time from enrollment to the end of the 2nd treatment cycle (each cycle is 28 days).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2026
- Study start1 Feb 2026 (estimated)
- Primary completion1 Feb 2028 (estimated)
- Study completion1 Feb 2030 (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.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Qianzhanruiji Enterprise Consulting Co., Ltd.
- Responsible party
- Principal investigator, Qian Jiang, Peking University People's Hospital
- Investigators
- Qian Jiang, Dr., Principal investigator, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07407660
- Sponsor's protocol ID
- 2025-h243
- Status last verified
- Feb 2026
- First submitted
- 27 Jan 2026
- Last updated
- 12 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Shortened Venetoclax Duration Based on Bone Marrow Blasts on Day 14 Versus Standard Therapy in Elderly or Frail Patients With Acute Myeloid Leukemia Treated With Azacitidine Plus Venetoclax: A Multicenter Prospective Randomized Controlled Study
- ClinicalTrials.gov ID
- NCT07407660
- Acronym / sponsor ID
- 2025-h243
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Venetoclax, Azacitidine, Early assessment, Treatment optimization, Acute myeloid leukemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Shanghai Qianzhanruiji Enterprise Consulting Co., Ltd.
- Study start
- 1 Feb 2026 (estimated)
- Primary completion
- 1 Feb 2028 (estimated)
- Study completion
- 1 Feb 2030 (estimated)
- First posted
- 12 Feb 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 study aims to compare the efficacy and safety of a shortened treatment course based on the bone marrow blast count on Day 14 versus standard treatment in patients with acute myeloid leukemia treated with venetoclax plus azacitidine.
Detailed description
The standard 28-day cycle of venetoclax is widely recommended for the first cycle of venetoclax plus azacitidine induction therapy in patients with acute myeloid leukemia (AML). However, it has been found that the 28-day treatment cycle was not tolerant for some patients due to severe myelosuppression and infection, which may lead to treatment interruption and delays in subsequent treatment cycles.
This is a multicenter, randomized controlled, open-label, non-inferiority study, which compare the efficacy and safety of a shortened treatment course based on the bone marrow blast count on Day 14 versus standard treatment in AML patients treated with venetoclax plus azacitidine induction therapy.
This study plans to enroll 250 newly diagnosed AML patients who are intolerant to intensive chemotherapy regimens. Enrolled subjects will be assigned to either the optimized treatment group or the standard treatment group in a 1:1 ratio with stratified blocked randomization, with ELN 2022 classification as the stratification factor. In the optimized treatment group, if the bone marrow blast count is <5% on Day 14 of the first induction, the duration of venetoclax will be shortened to 14 days; otherwise, the 28-day course will be completed as scheduled. In the standard treatment group, no bone marrow assessment will be performed on Day 14, and all patients will complete the 28-day treatment course. The duration of venetoclax in the second cycle will be 28 days or 21 days (if complete remission with incomplete hematologic recovery) for the two groups. The primary endpoint is the achievement of complete remission or complete remission with incomplete hematologic recovery (CR/CRi) within 2 treatment courses.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Optimized treatment | Experimental | Drug: Venetoclax, Drug: Azacitidine (AZA)Bone marrow aspiration is perfomed on day 14 of the first induction cycle. If the bone marrow blast count is <5%, the duration of venetoclax will be shortened to 14 days; otherwise, the 28-day course will be completed. In the second cycle, patients who fail to achieve CR/CRi and those who achieve CR will receive a 28-day course of venetoclax plus azacitidine; for patients who achieve CRi, a 21-day course of venetoclax plus azacitidine will be administered within 14 days following the first cycle. Treatment for subsequent cycles will be determined according to the investigators' local clinical practice, including but not limited to continued venetoclax-azacitidine therapy, switching to intensive chemotherapy, or allogeneic hematopoietic stem cell transplantation. |
| Standard treatment | Active comparator | Drug: Venetoclax, Drug: Azacitidine (AZA)No bone marrow assessment was performed on day 14 of the first induction cycle. All the patients receive the 28-day course of venetoclax plus azacitidine for the first cycle. In the second cycle, patients who fail to achieve CR/CRi and those who achieve CR will receive a 28-day course of venetoclax plus azacitidine; for patients who achieve CRi, a 21-day course of venetoclax plus azacitidine will be administered within 14 days following the first cycle. Treatment for subsequent cycles will be determined according to the investigators' local clinical practice, including but not limited to continued venetoclax-azacitidine therapy, switching to intensive chemotherapy, or allogeneic hematopoietic stem cell transplantation. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Patients with newly diagnosed acute myeloid leukemia who meet the WHO 2022 criteria.
- Meeting one of the following conditions:
- Received induction therapy with the azacitidine plus venetoclax regimen (azacitidine for injection: 75 mg/m² subcutaneously on Days 1-7; venetoclax tablets: 100 mg on Day 1, 200 mg on Day 2, and 400 mg once daily starting on Day 3) for 12-14 days. Dose adjustment of venetoclax shall be performed if combined with strong or moderate CYP3A/P-gp inhibitors.
- Completed risk stratification assessment per the ELN 2022 criteria.
- Signed the informed consent form.
Key exclusion criteria (10 listed)
- Diagnosis of acute promyelocytic leukemia, AML with t(8;21)(q22;q22.1)/ RUNX1::RUNX1T1 translocation, or blast crisis of chronic myeloid leukemia (CML).
- Prior treatment with venetoclax before the diagnosis of acute myeloid leukemia.
- A history of allogeneic hematopoietic stem cell transplantation.
- Severe hepatic or renal impairment, defined by the presence of any of the following abnormalities: aspartate aminotransferase (AST) > 2.5 × ULN; alanine aminotransferase (ALT) > 2.5 × ULN; creatinine clearance rate < 30 mL/min (calculated by the Cockcroft-Gault formula); or total bilirubin > 3 × ULN.
- Presence of acute active infection requiring intravenous systemic therapy.
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 |
|---|---|---|
| VenetoclaxTablet | Drug | Optimized treatment, Standard treatment |
| Azacitidine (AZA)Solution for subcutaneous | Drug | Optimized treatment, Standard treatment |
Primary outcome
Achievement of CR/CRi within 2 treatment cycles
Time frame: At the end of Cycle 1 and Cycle 2 (each cycle is 28 days). If CRi, repeat 2 weeks later.
6 secondary outcomes
Achievement of CR within 2 treatment cycles
Time frame: At the end of Cycle 1 and Cycle 2 (each cycle is 28 days). If CRi, repeat 2 weeks later.
Achievement of CR/CRi during treatment with the venetoclax plus azacitidine regimen
Time frame: At the end of Cycles 1, 2, 3, and 5 (each cycle is 28 days) of the venetoclax-azacitidine regimen, and every 2 cycles thereafter.
Achievement of MRD negativity during treatment with the venetoclax plus azacitidine regimen
Time frame: At the end of Cycles 1, 2, 3, and 5 (each cycle is 28 days) of the venetoclax-azacitidine regimen, and every 2 cycles thereafter.
Relapse-free survival
Time frame: From the first achievement of CR/CRi to disease relapse or death from any cause, whichever came first, assessed up to 48 months.
Overall survival
Time frame: Time from enrollment to death from any cause, assessed up to 48 months.
Adverse events
Time frame: Time from enrollment to the end of the 2nd treatment cycle (each cycle is 28 days).
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 12 Feb 2026.
- Individual participant dataSharing planned: Yes
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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.
Qian Jiang, Dr. principal investigator
Peking University People's Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Venetoclax and Azacitidine (AZA) in people with Acute Myeloid Leukemia, run by Peking University People's Hospital.
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 13 inclusion and 10 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 12 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 Qian Jiang, Mr. (+86-010-88326850, jiangqian@medmail.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Achievement of CR/CRi within 2 treatment cycles (time frame: At the end of Cycle 1 and Cycle 2 (each cycle is 28 days). If CRi, repeat 2 weeks later.). 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 12 Feb 2026; AzTrials last refreshed its copy on 6 Nov 2026.