Acute Myeloid LeukemiaPhase 2NCT06429098
Study of A Venetoclax-based, Anthracycline-free Regimen in Newly Diagnosed CBFβ::MYH11(+) AML
A Phase 2 study testing Venetoclax, azacitidine and decitabine in people with Acute Myeloid Leukemia, run by The First Affiliated Hospital of Soochow University at 2 sites in China.
Trial summary
This investigator-initiated, single-arm, phase II trial is aimed to evaluate the efficacy and safety of a venetoclax-based, anthracycline-free regimen in patients with newly diagnosed CBFβ::MYH11-positive acute myeloid leukemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Venetoclax, azacitidine and decitabine
- 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
- 2 sitesChina
- Study dates
- 1 Jan 2024to 31 Dec 2027
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults ≥ 18 years.
- Newly diagnosed CBFβ::MYH11(+) AML.
- Performance status 0-3 on the Eastern Cooperative Oncology Group (ECOG) Scale.
- Subject must voluntarily sign and date an informed consent, prior to the initiation of any screening or study-specific procedures.
- Ferrara's criteria are used to determine whether a patient is unfit, and a patient is deemed unfit if at least one of the following criteria is met:
- Age>75 years.
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- There are serious underlying heart, lung, kidney, liver complications.
- There are active infections that do not respond to anti-infective therapy.
- There is cognitive impairment.
- Other comorbidities that the doctor determines are not suitable for intensive chemotherapy.
Who cannot join
- Subject has received treatment with a hypomethylating agent and/or other chemotherapeutic agents either conventional or experimental or targeted drug therapy for AML (except oral hydroxyurea and/or leukocytometry to reduce white blood cell count).
- Pregnant or lactating women.
- To the knowledge of the subject and investigator, subject may not be able to complete all study visits or procedures required by the study protocol, including follow-up visits, and/or be unable to comply with the required study procedures.
- Other conditions deemed unsuitable for participation in this study by the investigator.
Trial locations and contacts
2 sites in 1 country.
China2 sites
Ethical Committee of the First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215000
The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology
Suzhou, Jiangsu, 215000
About this study
Official title: Study of A Venetoclax-based, Anthracycline-free Regimen in Patients With Newly Diagnosed CBFβ::MYH11-positive Acute Myeloid Leukemia
This investigator-initiated, single-arm, phase II trial is aimed to evaluate the efficacy and safety of a venetoclax-based, anthracycline-free regimen in patients with newly diagnosed CBFβ::MYH11-positive acute myeloid leukemia.
Read the detailed description
Primary Objectives:
To determine the CR (complete remission) / CRi (complete remission with incomplete blood count recovery) rate of 2 cycles of VEN/HMA in patients with newly diagnosed (ND) CBFβ::MYH11-positive acute myeloid leukemia(AML).
Secondary Objectives:
- To determine the overall response rate (ORR) of 2 cycles of VEN/HMA in patients with ND CBFβ::MYH11-positive AML.
- To determine the safety of the combination regimen.
- To study the trajectories of molecular measurable residual disease (MRD) during the therapy.
- To evaluate the impact of baseline genomic alterations on response and survival of the combination regimen.
- To assess the duration of response, overall survival (OS) and event free survival (EFS) of patients.
OUTLINE:
INDUCTION:
Patients with newly diagnosed CBFβ::MYH11(+) AML receive 2 cycles of VEN/HMA as induction therapy. Venetoclax orally (PO) once daily (QD) on days 1-28, azacitidine subcutaneously (SC) on days 1-7 or decitabine intravenously (IV) over 30-60 minutes on days 1-5.
CONSOLIDATION:
Patient fitness will be reassessed according to the Ferrara criteria if CR or CRi is achieved after 2 cycles of VEN/HMA. Fit patients will receive four cycles of consolidation therapy with high-dose cytarabine (2g/m2 every 12 hours, on days 1-3) combined with venetoclax (on days 1-7). Unfit patients will continue to receive VEN/HMA until disease progression.
After completion of study treatment, patients are followed up at 30 days and then every 3 months thereafter.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Venetoclax, azacitidine, decitabine, Cytarabine
- Keywords
- venetoclax, 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
Also called ABT-199, ABT199, Venclexta
Given PO, once daily. Treatment in cycle 1, the dose is 100 mg on day 1, then ramp up to 400mg. In all subsequent cycles, the dose of venetoclax is initiated at 400 mg daily.
azacitidineDrug
Also called 5 AZC, 5-AZC
Given SC
decitabineDrug
Also called Dacogen
Given IV
CytarabineDrug
Also called 1-.beta.-Cytosine arabinoside, Ara-C
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax, Azacitidine/Decitabine/, Cytarabine | Experimental | INDUCTION: Patients receive 2 cycles of VEN/HMA as induction therapy. Venetoclax orally (PO) once daily (QD) on days 1-28, azacitidine subcutaneously (SC) on days 1-7 or decitabine intravenously (IV) over 30-60 minutes on days 1-5.
CONSOLIDATION: Fit patients will receive four cycles of consolidation therapy with high-dose cytarabine (2g/m2 every 12 hours, on days 1-3) combined with venetoclax (on days 1-7). Unfit patients will continue to receive VEN/HMA until disease progression. Receives: Drug: Venetoclax; Drug: azacitidine; Drug: decitabine; Drug: Cytarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
composite complete remission rate
CR/CRi rate will be determined.
Measured: after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).
Secondary outcomes (7)
Overall response rate (ORR)
ORR will be determined.
Measured: after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).
Incidence of adverse events
Safety profile based on NCI CTCAE version 5.0 will be determined.
Measured: From the start of treatment until death or last follow-up, assessed for up to 3 years.
measurable residual disease (MRD) negativity
MRD will be assessed by real-time qRCR.
Measured: From the start of treatment until death or last follow-up, assessed for up to 3 years.
Impact of concurrent gene mutations
The impact of concurrent gene mutations ( analysis via an 81-gene institutional next-generation sequencing platform) on response and the survival of the combination regimen will be assessed.
Measured: Baseline
Overall survival (OS)
OS will be assessed.
Measured: From the start of treatment until death or last follow-up, assessed for up to 3 years.
Event-free survival (EFS)
EFS will be assessed.
Measured: up to 3 years.
Duration of response (DOR)
DOR will be assessed.
Measured: up to 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2024
- Study start1 Jan 2024
- Primary completion30 Jun 2027 (estimated)
- Study completion31 Dec 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 LeukemiaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ying Wang, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06429098
- Sponsor's protocol ID
- 2023371
- Status last verified
- Aug 2026
- First submitted
- 14 May 2024
- Last updated
- 9 Sep 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 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of A Venetoclax-based, Anthracycline-free Regimen in Patients With Newly Diagnosed CBFβ::MYH11-positive Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT06429098
- Acronym / sponsor ID
- 2023371
- Conditions
- Acute Myeloid Leukemia
- Keywords
- venetoclax, acute myeloid leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Jan 2024 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 24 May 2024 (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 investigator-initiated, single-arm, phase II trial is aimed to evaluate the efficacy and safety of a venetoclax-based, anthracycline-free regimen in patients with newly diagnosed CBFβ::MYH11-positive acute myeloid leukemia.
Detailed description
Primary Objectives:
To determine the CR (complete remission) / CRi (complete remission with incomplete blood count recovery) rate of 2 cycles of VEN/HMA in patients with newly diagnosed (ND) CBFβ::MYH11-positive acute myeloid leukemia(AML).
Secondary Objectives:
- To determine the overall response rate (ORR) of 2 cycles of VEN/HMA in patients with ND CBFβ::MYH11-positive AML.
- To determine the safety of the combination regimen.
- To study the trajectories of molecular measurable residual disease (MRD) during the therapy.
- To evaluate the impact of baseline genomic alterations on response and survival of the combination regimen.
- To assess the duration of response, overall survival (OS) and event free survival (EFS) of patients.
OUTLINE:
INDUCTION:
Patients with newly diagnosed CBFβ::MYH11(+) AML receive 2 cycles of VEN/HMA as induction therapy. Venetoclax orally (PO) once daily (QD) on days 1-28, azacitidine subcutaneously (SC) on days 1-7 or decitabine intravenously (IV) over 30-60 minutes on days 1-5.
CONSOLIDATION:
Patient fitness will be reassessed according to the Ferrara criteria if CR or CRi is achieved after 2 cycles of VEN/HMA. Fit patients will receive four cycles of consolidation therapy with high-dose cytarabine (2g/m2 every 12 hours, on days 1-3) combined with venetoclax (on days 1-7). Unfit patients will continue to receive VEN/HMA until disease progression.
After completion of study treatment, patients are followed up at 30 days and then every 3 months thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venetoclax, Azacitidine/Decitabine/, Cytarabine | Experimental | Drug: Venetoclax, Drug: azacitidine, Drug: decitabine, Drug: CytarabineINDUCTION: Patients receive 2 cycles of VEN/HMA as induction therapy. Venetoclax orally (PO) once daily (QD) on days 1-28, azacitidine subcutaneously (SC) on days 1-7 or decitabine intravenously (IV) over 30-60 minutes on days 1-5. CONSOLIDATION: Fit patients will receive four cycles of consolidation therapy with high-dose cytarabine (2g/m2 every 12 hours, on days 1-3) combined with venetoclax (on days 1-7). Unfit patients will continue to receive VEN/HMA until disease progression. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Adults ≥ 18 years.
- Newly diagnosed CBFβ::MYH11(+) AML.
- Performance status 0-3 on the Eastern Cooperative Oncology Group (ECOG) Scale.
- Subject must voluntarily sign and date an informed consent, prior to the initiation of any screening or study-specific procedures.
- Ferrara's criteria are used to determine whether a patient is unfit, and a patient is deemed unfit if at least one of the following criteria is met:
Key exclusion criteria (4 listed)
- Subject has received treatment with a hypomethylating agent and/or other chemotherapeutic agents either conventional or experimental or targeted drug therapy for AML (except oral hydroxyurea and/or leukocytometry to reduce white blood cell count).
- Pregnant or lactating women.
- To the knowledge of the subject and investigator, subject may not be able to complete all study visits or procedures required by the study protocol, including follow-up visits, and/or be unable to comply with the required study procedures.
- Other conditions deemed unsuitable for participation in this study by the investigator.
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 |
|---|---|---|
| VenetoclaxAlso: ABT-199, ABT199, VenclextaGiven PO, once daily. Treatment in cycle 1, the dose is 100 mg on day 1, then ramp up to 400mg. In all subsequent cycles, the dose of venetoclax is initiated at 400 mg daily. | Drug | Venetoclax, Azacitidine/Decitabine/, Cytarabine |
| azacitidineAlso: 5 AZC, 5-AZCGiven SC | Drug | Venetoclax, Azacitidine/Decitabine/, Cytarabine |
| decitabineAlso: DacogenGiven IV | Drug | Venetoclax, Azacitidine/Decitabine/, Cytarabine |
| CytarabineAlso: 1-.beta.-Cytosine arabinoside, Ara-CGiven IV | Drug | Venetoclax, Azacitidine/Decitabine/, Cytarabine |
Primary outcome
composite complete remission rate
Time frame: after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).
CR/CRi rate will be determined.
7 secondary outcomes
Overall response rate (ORR)
Time frame: after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).
Incidence of adverse events
Time frame: From the start of treatment until death or last follow-up, assessed for up to 3 years.
measurable residual disease (MRD) negativity
Time frame: From the start of treatment until death or last follow-up, assessed for up to 3 years.
Impact of concurrent gene mutations
Time frame: Baseline
Overall survival (OS)
Time frame: From the start of treatment until death or last follow-up, assessed for up to 3 years.
Event-free survival (EFS)
Time frame: up to 3 years.
Duration of response (DOR)
Time frame: up to 3 years.
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 9 Sep 2026.
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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.
Ying Wang principal investigator
The First Affiliated Hospital of Soochow University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Venetoclax, azacitidine and decitabine in people with Acute Myeloid Leukemia, run by The First Affiliated Hospital of Soochow University at 2 sites 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 4 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: under way, not looking for participants, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
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?
composite complete remission rate (time frame: after 2 cycles of induction therapy with VEN/HMA (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 9 Sep 2026.