Acute Myeloid LeukemiaPhase 2NCT07078591
Venetoclax-containing Therapy Combined With Microtransplant for Newly Diagnosed AML
A Phase 2 study testing Venetoclax, Azacitidine (AZA) and Daunorubicin in people with Acute Myeloid Leukemia, run by Beijing 302 Hospital at 1 site in China.
Trial summary
The purpose of this study is to evaluate whether HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cell (GPBMC) infusion with venetoclax-containing regimens (microtransplant, MST) could improve survival in adult patients with newly diagnosed acute myeloid leukemia (AML). 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 (AZA) and Daunorubicin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 20 Jun 2025to Jun 2031
- Phase
- Phase 2
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
- Age >=18 years, male or female, non-limited by race or ethnicity.
- No prior anti-acute leukemia treatment (including hypomethylators for leukemia or MDS) with the exception that prior hydroxyurea and/or leukapheresis are permitted.
- Confirmed acute myeloid leukemia in accordance with WHO criteria with a WBC count < 25 × 109/L.
- Adequate hepatic function including alanine transaminase (ALT) and aspartate aminotransferase (AST )<= 3 × upper limit of normal(ULN), and total bilirubin <= 1.5 × ULN.
- Adequate renal function including serum creatinine <= 2 × ULN or CrCl>= 40mL/min.
- LVEF measured by echocardiogram is within the normal range (LVEF > 50%).
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- The subject must have one donor who is >= 18 years old and HLA matched at 0-7/10 loci (i.e., at least 3 HLA loci must be mismatched). In addition, the donor voluntarily donates hematopoietic stem cells and signs the consent form.
- Each subject (or his/her legal representatives) must sign the Informed Consent Form (ICF), indicating that he/she understands the purpose and procedures of research, and is willing to participate in research.
- Donor inclusion criteria: The donor meets the institution's criteria for related peripheral blood hematopoietic stem cell donors. The donor must be able to tolerate the cell separation and collection process, and sign the Informed Consent Form.
Who cannot join
- Acute promyelocytic leukemia, myeloid sarcoma, chronic myeloid leukemia accelerated phase and blast crisis.
- Uncontrolled infection or hemorrhage.
- Cardiovascular disease with clinical significance, such as uncontrolled or highly symptomatic cardiac arrhythmias, congestive heart failure, or myocardial infarction within 6 months prior to screening, or New York Heart Association (NYHA) function class 3 (moderate) or class 4 (severe) heart disease.
- Uncontrolled autoimmune disease or requiring immunosuppression treatment.
- History of severe blood infusion reaction.
- Nursing women, women of childbearing potential with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception.
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- Psychiatric disorder or cognitive impairment that in the researcher's judgment would make the subject not likely to adhere to the protocol requirements.
- Any major surgery within 4 weeks before enrollment.
- Life-threatening illness other than AML or uncontrolled intercurrent illness.
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, 1 recruiting
- Recruiting
Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital
Beijing, 100071
Bo Cai, MD, +861066947168, caibo2008@163.com
Yangyang Lei, MD, +861066947180, 942056176@qq.com
About this study
Official title: Phase 2 Study of Venetoclax-containing Therapy in Combination With HLA-mismatched Mobilized Peripheral Blood Mononuclear Cell Infusion for Newly Diagnosed Acute Myeloid Leukemia
The purpose of this study is to evaluate whether HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cell (GPBMC) infusion with venetoclax-containing regimens (microtransplant, MST) could improve survival in adult patients with newly diagnosed acute myeloid leukemia (AML).
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Venetoclax, Azacitidine (AZA), Daunorubicin, Idarubicin（IDA）, Cytarabine (Ara-C)
- Keywords
- venetoclax, microtransplant, microtransplantation
- 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
Given PO
Azacitidine (AZA)Drug
Given SC
DaunorubicinDrug
Given IV
Idarubicin（IDA）Drug
Given IV
Cytarabine (Ara-C)Drug
Given IV
GPBMC infusionBiological
HLA-mismatched donor GPBMC infusion
| Study group | Role | What they receive |
|---|---|---|
| Participants fit for intensive therapy | Experimental | Patients fit for intensive therapy.
Induction:
Genetically low-risk:
Daunorubicin 60mg/m2 or idarubicin 12mg/m2 IV daily on Days 1-3; Cytarabine 100mg/m2 IV daily on Days 1-7; with or without Venetoclax 100mg on Day 4, 200mg on Day 5, 400mg on Days 6-11. Other induction regimens are also allowed. No more than two cycles. Continue to consolidation phase if achieving CR/CRi.
Genetically intermediate/high-risk:
Venetoclax 100mg on Day 1, 200mg on Day 2, 400 mg on Day 3-28; Azacitidine 75mg/m2 SC daily on Days 1-7. Other induction regimens are also allowed. No more than two cycles. Continue to consolidation phase if achieving CR/CRi and refusing allogeneic HSCT.
Consolidation:
Patients < 60 years:
Cytarabine 1.5g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles.
Patients ≥ 60 years:
Cytarabine 1g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles. Receives: Drug: Venetoclax; Drug: Azacitidine (AZA); Drug: Daunorubicin; Drug: Idarubicin（IDA）; Drug: Cytarabine (Ara-C); Biological: GPBMC infusion |
| Participants unfit for intensive therapy but fit for lower intensity therapy | Experimental | Patients unfit for intensive therapy but fit for lower intensity therapy at diagnosis according to Ferrara 2013 criteria.
Induction phase:
Venetoclax 100mg on Day 1, 200mg on Day 2, 400 mg on Days 3-28; Azacitidine 75mg/m2 SC daily on Days 1-7. 28 days per cycle, and no more than two cycles. Re-evaluate unfitness and continue to consolidation phase if achieving CR/CRi and refusing allogeneic HSCT.
Consolidation phase:
Convert to "fit" patients and < 60 years Cytarabine 1.5g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles.
Convert to "fit" patients and ≥ 60 years:
Cytarabine 1g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles.
Still "unfit" patients:
Venetoclax 400 mg on Days 1-14; Azacitidine 75mg/m2 SC daily on Days 1-7. Infuse donor GPBMCs on Day 15. 28 days per cycle until progression or untolerated. Receives: Drug: Venetoclax; Drug: Azacitidine (AZA); Drug: Cytarabine (Ara-C); Biological: GPBMC infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival (OS)
OS is defined as the number of days from the date of initiation of treatment to the date of death.
Measured: Measured up to 4 years after the last participant is enrolled
Secondary outcomes (2)
Event-free Survival (EFS)
EFS is defined as the number of days from initiation of treatment to the date of progressive disease, relapse from CR or CRi, treatment failure or death from any cause.
Measured: Measured up to 4 years after the last participant is enrolled
Non-relapse Mortality (NRM)
NRM is defined as the death without relapse.
Measured: Measured up to 4 years after the last participant is enrolled
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2025
- Study start20 Jun 2025
- Primary completionJun 2030 (estimated)
- Study completionJun 2031 (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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing 302 Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Bo Cai, MD, Principal investigator, Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07078591
- Sponsor's protocol ID
- VEN-MST for AML
- Status last verified
- Jul 2026
- First submitted
- 14 Jul 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No