Acute Myeloid Leukemia (AML)Phase 1/2NCT07451912
Venetoclax Plus Hypomethylating Agents and Subcutaneous Cytarabine for CEBPA-Mutated AML
A Phase 1/2 study testing Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients) in people with Acute Myeloid Leukemia (AML) and CEBPA Mutation, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
The goal of this clinical trial is to learn if a treatment combination-venetoclax plus hypomethylating agents (like azacitidine or decitabine) and low-dose cytarabine-works to treat adults with newly diagnosed CEBPA-mutated acute myeloid leukemia (AML) who can't tolerate intensive chemotherapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML), CEBPA Mutation, Unfit, New-diagnosis AML
- What is being tested?
- Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteChina
- Study dates
- 15 Sep 2025to Dec 2027
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients can participate in this study only if they meet all the following enrollment criteria:
- Aged over 18 years, male, or female who is neither pregnant nor lactating;
- Newly diagnosed acute myeloid leukemia (AML) with CEBPA mutation;
- ECOG performance status ≤ Grade 3;
- Capable of understanding and willing to participate in the study, and able to sign the informed consent form;
- Patients are judged as "unfit" according to the Ferrara criteria. A patient is considered "unfit" if they meet at least one of the following criteria:
Show all 9Show fewer
- Advanced age: > 75 years old;
- Presence of severe underlying comorbidities involving the heart, lungs, kidneys, or liver; ③ Presence of active infection unresponsive to anti-infective treatment;
- Presence of cognitive impairment; ⑤ Poor performance status (persistent ECOG score ≥ Grade 3); ⑥ Other comorbidities judged by the investigator to make the patient unfit for intensive chemotherapy.
Who cannot join
- Patients are not suitable for participating in this study if they meet any of the following exclusion criteria:
- Pregnant or lactating women;
- Previous receipt of chemotherapy or targeted drug therapy for leukemia before the study treatment (except oral hydroxyurea used to reduce white blood cell count and/or leukapheresis);
- As known to the participant and the investigator, the participant may be unable to complete all study visits or procedures required by the study protocol (including follow-up visits) and/or unable to comply with the required study procedures;
- Other conditions judged by the investigator to make the patient unfit for participating in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Wenzhi Cai contact
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
The First Affiliated Hospital of Soochow University, 0086-0512-67972861, sdfyec@163.com
About this study
Official title: Prospective Multicenter Clinical Study of Venetoclax Combined With Hypomethylating Agents and Subcutaneous Cytarabine in Induction Therapy for CEBPA-Mutated Acute Myeloid Leukemia
The goal of this clinical trial is to learn if a treatment combination-venetoclax plus hypomethylating agents (like azacitidine or decitabine) and low-dose cytarabine-works to treat adults with newly diagnosed CEBPA-mutated acute myeloid leukemia (AML) who can't tolerate intensive chemotherapy. It will also check how safe this treatment combination is and explore how the disease might change if it comes back. The main questions it aims to answer are:
- How well does this treatment combination prevent the disease from coming back (relapse-free survival)?
- What percentage of participants achieve a good response (complete remission or complete remission with incomplete blood cell recovery) after 2 treatment cycles?
- What percentage of participants have no detectable remaining leukemia cells (measurable residual disease, MRD) after treatment? What side effects do participants have, and how serious are these side effects?
Read more
Participants will:
- First, go through a 2-cycle "induction phase": Take venetoclax by mouth (100mg on day 1, 200mg on day 2, 400mg from day 3 to day 28), get hypomethylating agents (azacitidine injected under the skin or decitabine injected into a vein), and low-dose cytarabine (injected under the skin) as planned.
- If they respond well to induction treatment, move to a "consolidation phase" and receive at least 4 more cycles of the same treatment combination.
- Have regular check-ups during treatment (like blood tests, bone marrow tests, and heart checks) to monitor treatment response and side effects.
- Be followed up for 2 years after treatment ends to check if the disease comes back and their overall health.
- Being tested
- Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients)
- 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.
Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients)Drug
- Induction Therapy (2 Cycles in Total) All medications are administered in a 28-day cycle; the second cycle is given regardless of the response to the first cycle. Medication Generic Name Dosage Form Dosage Administration Frequency Duration per Cycle Venetoclax Oral tablets 100 mg on Day 1; 200 mg on Day 2; 400 mg from Day 3 to Day 28 Once daily (oral) Days 1-28 Azacitidine (alternative to decitabine) Injectable 75 mg/m² per day Once daily (subcutaneous injection) Days 1-7 Decitabine (alternative to azacitidine) Injectable 20 mg/m² per day Once daily (intravenous injection) Days 1-5 Cytarabine (low-dose) Injectable 20 mg/m² per day Once daily (subcutaneous injection) Days 1-10
- Consolidation Therapy (At Least 4 Cycles) Same as the therapy of induction
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients) | Experimental | 1. Induction Therapy (2 Cycles in Total) All medications are administered in a 28-day cycle; the second cycle is given regardless of the response to the first cycle.
Medication Generic Name Dosage Form Dosage Administration Frequency Duration per Cycle Venetoclax Oral tablets 100 mg on Day 1; 200 mg on Day 2; 400 mg from Day 3 to Day 28 Once daily (oral) Days 1-28 Azacitidine (alternative to decitabine) Injectable 75 mg/m² per day Once daily (subcutaneous injection) Days 1-7 Decitabine (alternative to azacitidine) Injectable 20 mg/m² per day Once daily (intravenous injection) Days 1-5 Cytarabine (low-dose) Injectable 20 mg/m² per day Once daily (subcutaneous injection) Days 1-10
2. Consolidation Therapy (At Least 4 Cycles) Same as the therapy of induction Receives: Drug: Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Relapse-Free Survival (RFS)
Defined as the time from the date of achieving complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) to the first occurrence of disease relapse (reappearance of ≥5% blasts in the bone marrow, presence of blasts in the peripheral blood, or new extramedullary disease) or death from any cause, whichever comes first. For participants who remain in remission at the end of the follow-up period, RFS is censored at the date of the last confirmed remission assessment.
Measured: From date of achieving CR/CRi until first occurrence of disease relapse or death from any cause, assessed up to 48 months (2 years after the last patient has been enrolled into the study).
Secondary outcomes (5)
Composite Remission Rate after 2 Induction Cycles
Proportion of participants who achieve CR or CRi after completing 2 cycles of induction therapy. (Each cycle is 28 days) CR: Defined by the following criteria simultaneously: bone marrow blasts <5%, no blasts in the peripheral blood, absolute neutrophil count >1.0×10⁹/L, platelet count >100×10⁹/L, no extramedullary infiltration, and no relapse within 4 weeks.
CRi: Meets all CR criteria except for incomplete hematologic recovery (absolute neutrophil count ≤1.0×10⁹/L or platelet count ≤100×10⁹/L).
Measured: Proportion of participants who achieve CR or CRi after completing 2 cycles of induction therapy. (Each cycle is 28 days)
Minimal Residual Disease (MRD) Negativity Rate
Proportion of participants with MRD <1×10-⁴ detected by flow cytometry after treatment.
Measured: 21 to 28 days after the end of every treatment
Duration of Remission (DOR)
If no relapse or death occurs during follow-up, DOR is censored at the last follow-up date.
Measured: From date of achieving CR/CRi until first occurrence of disease relapse or death from any cause, assessed up to 60 months.
Relapse Rate
Proportion of participants who experience disease relapse after achieving CR/CRi during the study period.
Measured: Proportion of participants who experience disease relapse after achieving CR/CRi, assessed up to 48 months (2 years after the last patient has been enrolled into the study).
Overall Survival (OS)
Time from the start of treatment to death from any cause. For participants who remain alive at the end of the follow-up period, OS is censored at the date of the last survival confirmation.
Measured: From start of treatment until death from any cause, assessed up to 48 months (2 years after the last patient has been enrolled into the study).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2026
- Study start15 Sep 2025
- Primary completionSep 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chen Suning, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07451912
- Sponsor's protocol ID
- SZAML-05
- Status last verified
- Mar 2026
- First submitted
- 15 Dec 2025
- Last updated
- 5 Mar 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Multicenter Clinical Study of Venetoclax Combined With Hypomethylating Agents and Subcutaneous Cytarabine in Induction Therapy for CEBPA-Mutated Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07451912
- Acronym / sponsor ID
- SZAML-05
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 29 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 15 Sep 2025 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 5 Mar 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.
The goal of this clinical trial is to learn if a treatment combination-venetoclax plus hypomethylating agents (like azacitidine or decitabine) and low-dose cytarabine-works to treat adults with newly diagnosed CEBPA-mutated acute myeloid leukemia (AML) who can't tolerate intensive chemotherapy. It will also check how safe this treatment combination is and explore how the disease might change if it comes back. The main questions it aims to answer are:
- How well does this treatment combination prevent the disease from coming back (relapse-free survival)?
- What percentage of participants achieve a good response (complete remission or complete remission with incomplete blood cell recovery) after 2 treatment cycles?
- What percentage of participants have no detectable remaining leukemia cells (measurable residual disease, MRD) after treatment? What side effects do participants have, and how serious are these side effects?
Participants will:
- First, go through a 2-cycle "induction phase": Take venetoclax by mouth (100mg on day 1, 200mg on day 2, 400mg from day 3 to day 28), get hypomethylating agents (azacitidine injected under the skin or decitabine injected into a vein), and low-dose cytarabine (injected under the skin) as planned.
- If they respond well to induction treatment, move to a "consolidation phase" and receive at least 4 more cycles of the same treatment combination.
- Have regular check-ups during treatment (like blood tests, bone marrow tests, and heart checks) to monitor treatment response and side effects.
- Be followed up for 2 years after treatment ends to check if the disease comes back and their overall health.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients) | Experimental | Drug: Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients)1. Induction Therapy (2 Cycles in Total) All medications are administered in a 28-day cycle; the second cycle is given regardless of the response to the first cycle. Medication Generic Name Dosage Form Dosage Administration Frequency Duration per Cycle Venetoclax Oral tablets 100 mg on Day 1; 200 mg on Day 2; 400 mg from Day 3 to Day 28 Once daily (oral) Days 1-28 Azacitidine (alternative to decitabine) Injectable 75 mg/m² per day Once daily (subcutaneous injection) Days 1-7 Decitabine (alternative to azacitidine) Injectable 20 mg/m² per day Once daily (intravenous injection) Days 1-5 Cytarabine (low-dose) Injectable 20 mg/m² per day Once daily (subcutaneous injection) Days 1-10 2. Consolidation Therapy (At Least 4 Cycles) Same as the therapy of induction |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients can participate in this study only if they meet all the following enrollment criteria:
Key exclusion criteria (5 listed)
- Patients are not suitable for participating in this study if they meet any of the following exclusion criteria:
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 |
|---|---|---|
| Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients)1. Induction Therapy (2 Cycles in Total) All medications are administered in a 28-day cycle; the second cycle is given regardless of the response to the first cycle. Medication Generic Name Dosage Form Dosage Administration Frequency Duration per Cycle Venetoclax Oral tablets 100 mg on Day 1; 200 mg on Day 2; 400 mg from Day 3 to Day 28 Once daily (oral) Days 1-28 Azacitidine (alternative to decitabine) Injectable 75 mg/m² per day Once daily (subcutaneous injection) Days 1-7 Decitabine (alternative to azacitidine) Injectable 20 mg/m² per day Once daily (intravenous injection) Days 1-5 Cytarabine (low-dose) Injectable 20 mg/m² per day Once daily (subcutaneous injection) Days 1-10 2. Consolidation Therapy (At Least 4 Cycles) Same as the therapy of induction | Drug | Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients) |
Primary outcome
Relapse-Free Survival (RFS)
Time frame: From date of achieving CR/CRi until first occurrence of disease relapse or death from any cause, assessed up to 48 months (2 years after the last patient has been enrolled into the study).
Defined as the time from the date of achieving complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) to the first occurrence of disease relapse (reappearance of ≥5% blasts in the bone marrow, presence of blasts in the peripheral blood, or new extramedullary disease) or death from any cause, whichever comes first. For participants who remain in remission at the end of the follow-up period, RFS is censored at the date of the last confirmed remission assessment.
5 secondary outcomes
Composite Remission Rate after 2 Induction Cycles
Time frame: Proportion of participants who achieve CR or CRi after completing 2 cycles of induction therapy. (Each cycle is 28 days)
Minimal Residual Disease (MRD) Negativity Rate
Time frame: 21 to 28 days after the end of every treatment
Duration of Remission (DOR)
Time frame: From date of achieving CR/CRi until first occurrence of disease relapse or death from any cause, assessed up to 60 months.
Relapse Rate
Time frame: Proportion of participants who experience disease relapse after achieving CR/CRi, assessed up to 48 months (2 years after the last patient has been enrolled into the study).
Overall Survival (OS)
Time frame: From start of treatment until death from any cause, assessed up to 48 months (2 years after the last patient has been enrolled into the study).
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 5 Mar 2026.
Related trials for Acute Myeloid Leukemia (AML)
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
Wenzhi Cai contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients) in people with Acute Myeloid Leukemia (AML) and CEBPA Mutation, run by The First Affiliated Hospital of Soochow University 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 9 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 5 Mar 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Suning Chen (008613814881746, chensuning@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relapse-Free Survival (RFS) (time frame: From date of achieving CR/CRi until first occurrence of disease relapse or death from any cause, assessed up to 48 months (2 years after the last patient has been enrolled into the study).). 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 5 Mar 2026.