Leukemia, Myeloid, AcuteNCT05471700
Single Arm Study of Azacitidine and Venetoclax for Treatment of Newly Diagnosed Fit Acute Myeloid Leukemia Patients
A study testing Azacitidine and Venetoclax in people with Leukemia, Myeloid, Acute, run by Zhujiang Hospital at 1 site in China.
Trial summary
The goal of this clinical research study is to evaluate the safety and efficacy of Azacytidine in combination with Venetoclax in patients with newly diagnosed fit acute myeloid leukemia. Read more about this study
- Who is it for?
- Ages 45 to 65 years
- Conditions the study is for
- Leukemia, Myeloid, Acute
- What is being tested?
- Azacitidine and Venetoclax
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2022to 4 Apr 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 45 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1.Age 45 to 65 years old,both male and female
- 2.Be able to understand and sign informed consent
- 3.Untreated AML patients (non-APL) diagnosed according to the 2016 World Health Organization (WHO) classification and diagnostic criteria for acute myeloid leukemia
- 4.Patients with an ECOG performance status 0,1,2 or 3
- 5.Expected survival time≥3 months
- 6.Laboratory indicators meet the following standards:
- Bilrubin, ALT and AST were all less than 3 times the upper limit of normal within 7 days before the first day of treatment
- Serum creatinine clearance rate is greater than 30ml/min
Who cannot join
- 1.patients participating in other interventional or observational clinical studies currently
- 2.Patients has a history of myeloproliferative neoplasm [MPN]
- 3.Patients with acute promyelocytic leukemia
- 4.Active CNS involvement in patients with acute myeloid leukemia
- 5.Patients with active hepatitis B or C, HIV infection before enrollment
- 6.Cardiovascular status of patients evaluate by NYHA classification method > 2
Show all 12Show fewer
- 7.Patients have chronic respiratory disease requiring continuous oxygen treatment, or any sitiuation investigator believes will adversely affect this study (including renal, neurological, psychiatric, endocrine, metabolic, immune, hepatic, cardiovascular disease and known history of allergy to any study drug)
- 8.Patients suffering from malabsorption syndrome or other diseases that prevent the enteral route of administration
- 9.patients present with an uncontrolled systemic infection (viral, bacterial, or fungal)
- 10.Patients with a history of other malignancies within 2 years before enrollment, except for the following cases:
- Adequately treated carcinoma in situ of the cervix or carcinoma of the breast
- Basal cell carcinoma or localized squamous cell carcinoma of skin
Trial locations and contacts
1 site in 1 country.
China1 site
Nanfang Hospital of Southern Medical University
Guanzhou, Guandong, 510250
About this study
Official title: To Evaluate Safety and Efficacy of Azacitidine Combination With Venetoclax for Treatment of Newly Diagnosed Fit Acute Myeloid Leukemia Patients：A Multicenter, Single-arm Study
The goal of this clinical research study is to evaluate the safety and efficacy of Azacytidine in combination with Venetoclax in patients with newly diagnosed fit acute myeloid leukemia.
Read the detailed description
The Study Drug:
Azacitidine is a DNA Demethylated drug, which is approvd for treatment of elderly AML patients Venetoclax targets the anti-apoptotic protein Bcl-2 and the process of oxidative phosphorylation to induce apoptosis of leukemia cells and exhibits anti-tumor activity.
Study Group:
single arm: AZA plus Venetoclax
Study Drug Administration:
Each course is 28 days long. Venetoclax is taken once daily by oral. The doses of Venetoclax is 100 mg on Day 1, 200 mg on Day 2, reaching a target dose of 400 mg on Day 3 and continuing through Day 28. If concomitantly receiving azoles, the dose of Venetoclax will be adjusted according to the instructions. Azacitidine is taken 75mg/m2 on Days 1-7. Subjects would received 1-2 cycles induction therapy. Once get remission, subjects would received the same indution therapy of middle to high dose of cytarabine based chemotherapy at the discretion of the physician for consolidation. Allo-HSCT is suggested for subjects with the moderate to adverse ELN2017 risk.
Study Visit:
After treatment, relapse and survival will be followed monthly by telephone, on-site visits, and/or study medical records for up to two years (calculated from the first day of induction therapy); Thereafter follow-up every 3 months for one year.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- Azacitidine, Venetoclax
- Keywords
- Acute Myeloid Leukemia, Newly Diagnosed AML, Azacitidine, Venetoclax, Induction Therapy
- 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.
AzacitidineDrug
Also called 5-Azacitidine, 5-aza
Subjects will receive Azacitidine 75 mg/m2 on days 1-7.Each course is 28 days long.
VenetoclaxDrug
Also called Venclexta, Venclyxto
Subjects would receive Venetoclax once daily by oral. The doses of Venetoclax is 100 mg on Day 1, 200 mg on Day 2, reaching a target dose of 400 mg on Day 3 and continuing through Day 28. Each course is 28 days long.
| Study group | Role | What they receive |
|---|---|---|
| Azacitidine combined with Venetoclax | Experimental | Each course is 28 days long. Subjects with newly diagnosed AML receive Venetoclax once daily by oral. The doses of Venetoclax is 100 mg on Day 1, 200 mg on Day 2, reaching a target dose of 400 mg on Day 3 and continuing through Day 28. If concomitantly receiving azoles, the dose of Venetoclax will be adjusted according to the instructions. At the same time patients will receive Azacitidine 75mg/m2 subcutaneous on Days 1-7.The induction therapy includes 1-2 cycle until subjects get remission.
Once complete remission, subjects will receive consolidation. Azacitidine combined with Venetoclax or middle to high dose of arabinoside based chemotherapy would be given at the discretion of the physician. Receives: Drug: Azacitidine; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
ORR is defined as the percentage of participants who achieve the CR + CRi + Partial Remission (PR) as evaluated by the investigator.
Measured: Up to 36 months
Secondary outcomes (5)
Minimal residual disease (MRD) negative response rate
The number of subjects who obtained MRD negative as a percentage of all evaluable subjects.
Measured: Up to 36 months
Duration of Remission (DOR)
From the first achievement of PR, CR, or CRi to time of disease progression
Measured: Up to 36 months
Allogeneic Hematopoietic Stem Cell Transplantation Rate
Proportion of subsequent allogeneic hematopoietic stem cell transplantation in intermediate-risk and adverse-risk patients
Measured: Up to 36 months
Overall Survival (OS)
From start of protocol treatment to time of death due to any cause, or until last follow-up
Measured: Up to 36 months
Event-Free Survival (EFS)
From start of protocol treatment to time of disease relapse/progression or death due to any cause, whichever occurs earlier; or until last follow-up
Measured: Up to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2022
- Study start1 Sep 2022
- Primary completion4 Apr 2023
- Study completion4 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- More trials run by Zhujiang 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zhujiang Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yuhua Li, Doctor, Principal investigator, Zhujiang Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05471700
- Sponsor's protocol ID
- AZA+Venetoclax
- Status last verified
- Jul 2025
- First submitted
- 1 Jul 2022
- Last updated
- 23 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: We will share our data within 6 months after the end of the trial. You can ask us for data or obtain it on the Clinical Trial Management Public Platfcrm of clinical trial. Supporting Information: Study Protocol
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- To Evaluate Safety and Efficacy of Azacitidine Combination With Venetoclax for Treatment of Newly Diagnosed Fit Acute Myeloid Leukemia Patients：A Multicenter, Single-arm Study
- ClinicalTrials.gov ID
- NCT05471700
- Acronym / sponsor ID
- AZA+Venetoclax
- Conditions
- Leukemia, Myeloid, Acute
- Keywords
- Acute Myeloid Leukemia, Newly Diagnosed AML, Azacitidine, Venetoclax, Induction Therapy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Zhujiang HospitalUniversity, hospital or other
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 4 Apr 2023 (actual)
- Study completion
- 4 Apr 2023 (actual)
- First posted
- 25 Jul 2022 (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.
The goal of this clinical research study is to evaluate the safety and efficacy of Azacytidine in combination with Venetoclax in patients with newly diagnosed fit acute myeloid leukemia.
Detailed description
The Study Drug:
Azacitidine is a DNA Demethylated drug, which is approvd for treatment of elderly AML patients Venetoclax targets the anti-apoptotic protein Bcl-2 and the process of oxidative phosphorylation to induce apoptosis of leukemia cells and exhibits anti-tumor activity.
Study Group:
single arm: AZA plus Venetoclax
Study Drug Administration:
Each course is 28 days long. Venetoclax is taken once daily by oral. The doses of Venetoclax is 100 mg on Day 1, 200 mg on Day 2, reaching a target dose of 400 mg on Day 3 and continuing through Day 28. If concomitantly receiving azoles, the dose of Venetoclax will be adjusted according to the instructions. Azacitidine is taken 75mg/m2 on Days 1-7. Subjects would received 1-2 cycles induction therapy. Once get remission, subjects would received the same indution therapy of middle to high dose of cytarabine based chemotherapy at the discretion of the physician for consolidation. Allo-HSCT is suggested for subjects with the moderate to adverse ELN2017 risk.
Study Visit:
After treatment, relapse and survival will be followed monthly by telephone, on-site visits, and/or study medical records for up to two years (calculated from the first day of induction therapy); Thereafter follow-up every 3 months for one year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Azacitidine combined with Venetoclax | Experimental | Drug: Azacitidine, Drug: VenetoclaxEach course is 28 days long. Subjects with newly diagnosed AML receive Venetoclax once daily by oral. The doses of Venetoclax is 100 mg on Day 1, 200 mg on Day 2, reaching a target dose of 400 mg on Day 3 and continuing through Day 28. If concomitantly receiving azoles, the dose of Venetoclax will be adjusted according to the instructions. At the same time patients will receive Azacitidine 75mg/m2 subcutaneous on Days 1-7.The induction therapy includes 1-2 cycle until subjects get remission. Once complete remission, subjects will receive consolidation. Azacitidine combined with Venetoclax or middle to high dose of arabinoside based chemotherapy would be given at the discretion of the physician. |
Who may qualify
- Ages
- 45 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 1.Age 45 to 65 years old,both male and female
- 2.Be able to understand and sign informed consent
- 3.Untreated AML patients (non-APL) diagnosed according to the 2016 World Health Organization (WHO) classification and diagnostic criteria for acute myeloid leukemia
- 4.Patients with an ECOG performance status 0,1,2 or 3
- 5.Expected survival time≥3 months
Key exclusion criteria (12 listed)
- 1.patients participating in other interventional or observational clinical studies currently
- 2.Patients has a history of myeloproliferative neoplasm [MPN]
- 3.Patients with acute promyelocytic leukemia
- 4.Active CNS involvement in patients with acute myeloid leukemia
- 5.Patients with active hepatitis B or C, HIV infection before enrollment
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 |
|---|---|---|
| AzacitidineAlso: 5-Azacitidine, 5-azaSubjects will receive Azacitidine 75 mg/m2 on days 1-7.Each course is 28 days long. | Drug | Azacitidine combined with Venetoclax |
| VenetoclaxAlso: Venclexta, VenclyxtoSubjects would receive Venetoclax once daily by oral. The doses of Venetoclax is 100 mg on Day 1, 200 mg on Day 2, reaching a target dose of 400 mg on Day 3 and continuing through Day 28. Each course is 28 days long. | Drug | Azacitidine combined with Venetoclax |
Primary outcome
Overall Response Rate (ORR)
Time frame: Up to 36 months
ORR is defined as the percentage of participants who achieve the CR + CRi + Partial Remission (PR) as evaluated by the investigator.
5 secondary outcomes
Minimal residual disease (MRD) negative response rate
Time frame: Up to 36 months
Duration of Remission (DOR)
Time frame: Up to 36 months
Allogeneic Hematopoietic Stem Cell Transplantation Rate
Time frame: Up to 36 months
Overall Survival (OS)
Time frame: Up to 36 months
Event-Free Survival (EFS)
Time frame: Up to 36 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2025.
- Individual participant dataSharing planned: Yes. We will share our data within 6 months after the end of the trial. You can ask us for data or obtain it on the Clinical Trial Management Public Platfcrm of clinical trial. Supporting Information: Study Protocol
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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. This study is not recruiting at the moment.
Yuhua Li, Doctor principal investigator
Zhujiang Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Azacitidine and Venetoclax in people with Leukemia, Myeloid, Acute, run by Zhujiang Hospital 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 8 inclusion and 12 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: finished, as of the sponsor's last update on 23 Sep 2025. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) (time frame: Up to 36 months). 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 23 Sep 2025.