Myelodysplastic SyndromesPhase 2NCT07238686
Venetoclax-containing Therapy Combined With Microtransplant for Intermediate-risk and Higher MDS
A Phase 2 study testing Venetoclax, Azacitidine (AZA) or Decitabine (DAC) and GPBMC infusion in people with Myelodysplastic Syndromes, run by Beijing 302 Hospital at 1 site in China.
Trial summary
This study aims to evaluate the safety and efficacy of a Venetoclax and hypomethylating agent-based regimen combined with infusion of HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cells (GPBMC) in patients with intermediate-risk and higher myelodysplastic syndromes who are ineligible for allogeneic… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Venetoclax, Azacitidine (AZA) or Decitabine (DAC) and GPBMC infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 20 Jun 2025to Jun 2031
- 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
- Age >=18 years, male or female, non-limited by race or ethnicity.
- Confirmed diagnosis of MDS according to the World Health Organization (WHO) 5th edition classification, based on histopathology and cytogenetics.
- Risk stratification according to the Revised International Prognostic Scoring System (IPSS-R) must place the patient in the intermediate-, high-, or very high-risk category.
- Not candidates for or refuse allogeneic hematopoietic stem cell transplantation.
- 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.
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- LVEF measured by echocardiogram is within the normal range (LVEF > 50%).
- 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
- 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.
- Psychiatric disorder or cognitive impairment that in the researcher's judgment would make the subject not likely to adhere to the protocol requirements.
- Major surgery within 4 weeks prior to enrollment.
- Life-threatening illness other than MDS 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
Fei Peng, MD, +861066947180, pengfei0118@foxmail.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 Intermediate-risk and Higher Myelodysplastic Syndromes
This study aims to evaluate the safety and efficacy of a Venetoclax and hypomethylating agent-based regimen combined with infusion of HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cells (GPBMC) in patients with intermediate-risk and higher myelodysplastic syndromes who are ineligible for allogeneic hematopoietic stem cell transplantation.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Venetoclax, Azacitidine (AZA) or Decitabine (DAC), GPBMC infusion
- Keywords
- venetoclax, hypomethylating agent, GPBMC infusion, microtransplant, microtransplantation
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VenetoclaxDrug
Given PO. For the first cycle, dose of 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Days 3-14. For other cycles, dose of 400 mg on Days 1-14. Note: The dose should be reduced to 100-200 mg daily if concomitant azole antifungals are required.
Azacitidine (AZA) or Decitabine (DAC)Drug
AZA: Given SC. Dose of 75 mg/m² on Days 1-7 of each cycle. DAC: Given IV. Dose of 20 mg/m² on Days 1-5 of each cycle.
GPBMC infusionBiological
HLA-mismatched donor GPBMCs are infused on Day 15.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | This cohort includes patients with intermediate-risk and higher MDS who are ineligible for or refuse allogeneic HSCT. Patients receive Venetoclax in combination with hypomethylating agent and HLA-mismatched donor GPBMC infusion. Treatment cycles are 28 days, repeated until disease progression or unacceptable toxicity. Receives: Drug: Venetoclax; Drug: Azacitidine (AZA) or Decitabine (DAC); 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 study entry to the date of death.
Measured: Measured up to 4 years after the last participant is enrolled
Overall response rate (ORR)
ORR is defined as complete remission (CR) (or CR equivalent) + partial remission (PR) + CR with limited count recovery (CRL) + CR with partial hematologic recovery (CRh) + hematologic improvement (HI) according to IWG 2023 criteria.
Measured: Measured up to 2 years after the last participant is enrolled
Secondary outcomes (7)
Modified overall response (mOR)
Modified OR is defined as CR+PR+CRL+CRh.
Measured: Measured up to 2 years after the last participant is enrolled
Complete remission (CR)
CR is defined as absolute neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, Hb ≥10 g/dL, bone marrow with < 5% blasts, and PB blasts 0%.
Measured: Measured up to 2 years after the last participant is enrolled
Partial remission (PR)
PR is defined as decrease of at least 50% in the percentage of blasts still ≥5% in the bone marrow, peripheral blood neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, Hb ≥10 g/dL.
Measured: Measured up to 2 years after the last participant is enrolled
CR with limited count recovery (CRL)
CRL is defined as bone marrow with less than 5% blasts, PB blasts 0%, and PB only meeting 1 or 2 of the following: absolute neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, Hb ≥10 g/dL.
Measured: Measured up to 2 years after the last participant is enrolled
CR with partial hematologic recovery (CRh)
CRh is defined as bone marrow with less than 5% blasts, and PB not meeting criteria for CR or CRL, no Hb threshold required, platelets ≥50 × 109/L, neutrophils ≥0.5 × 109/L, blasts 0%.
Measured: Measured up to 2 years after the last participant is enrolled
Progression-free survival (PFS)
PFS is defined as the number of days from the date of study entry to the date of the first of the following events: progressed disease; relapse from CR (or CR equivalent), PR, CRL, CRh, or HI; death from any cause.
Measured: Measured up to 4 years after the last participant is enrolled
Treatment-related mortality (TRM)
TRM is defined as the death related to treatment instead of disease progression.
Measured: Measured up to 2 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 posted20 Nov 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 Myelodysplastic SyndromesStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 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
- NCT07238686
- Sponsor's protocol ID
- VEN-MST for MDS
- Status last verified
- Jul 2026
- First submitted
- 16 Nov 2025
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Venetoclax-containing Therapy in Combination With HLA-mismatched Mobilized Peripheral Blood Mononuclear Cell Infusion for Intermediate-risk and Higher Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT07238686
- Acronym / sponsor ID
- VEN-MST for MDS
- Conditions
- Myelodysplastic Syndromes
- Keywords
- venetoclax, hypomethylating agent, GPBMC infusion, microtransplant, microtransplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Beijing 302 HospitalUniversity, hospital or other
- Study start
- 20 Jun 2025 (actual)
- Primary completion
- Jun 2030 (estimated)
- Study completion
- Jun 2031 (estimated)
- First posted
- 20 Nov 2025 (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 evaluate the safety and efficacy of a Venetoclax and hypomethylating agent-based regimen combined with infusion of HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cells (GPBMC) in patients with intermediate-risk and higher myelodysplastic syndromes who are ineligible for allogeneic hematopoietic stem cell transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: Venetoclax, Drug: Azacitidine (AZA) or Decitabine (DAC), Biological: GPBMC infusionThis cohort includes patients with intermediate-risk and higher MDS who are ineligible for or refuse allogeneic HSCT. Patients receive Venetoclax in combination with hypomethylating agent and HLA-mismatched donor GPBMC infusion. Treatment cycles are 28 days, repeated until disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age >=18 years, male or female, non-limited by race or ethnicity.
- Confirmed diagnosis of MDS according to the World Health Organization (WHO) 5th edition classification, based on histopathology and cytogenetics.
- Risk stratification according to the Revised International Prognostic Scoring System (IPSS-R) must place the patient in the intermediate-, high-, or very high-risk category.
- Not candidates for or refuse allogeneic hematopoietic stem cell transplantation.
- Adequate hepatic function including alanine transaminase (ALT) and aspartate aminotransferase (AST )<= 3 × upper limit of normal(ULN), and total bilirubin <= 1.5 × ULN.
Key exclusion criteria (8 listed)
- 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.
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 |
|---|---|---|
| VenetoclaxGiven PO. For the first cycle, dose of 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Days 3-14. For other cycles, dose of 400 mg on Days 1-14. Note: The dose should be reduced to 100-200 mg daily if concomitant azole antifungals are required. | Drug | Cohort 1 |
| Azacitidine (AZA) or Decitabine (DAC)AZA: Given SC. Dose of 75 mg/m² on Days 1-7 of each cycle. DAC: Given IV. Dose of 20 mg/m² on Days 1-5 of each cycle. | Drug | Cohort 1 |
| GPBMC infusionHLA-mismatched donor GPBMCs are infused on Day 15. | Biological | Cohort 1 |
Primary outcome
Overall survival (OS)
Time frame: Measured up to 4 years after the last participant is enrolled
OS is defined as the number of days from the date of study entry to the date of death.
Overall response rate (ORR)
Time frame: Measured up to 2 years after the last participant is enrolled
ORR is defined as complete remission (CR) (or CR equivalent) + partial remission (PR) + CR with limited count recovery (CRL) + CR with partial hematologic recovery (CRh) + hematologic improvement (HI) according to IWG 2023 criteria.
7 secondary outcomes
Modified overall response (mOR)
Time frame: Measured up to 2 years after the last participant is enrolled
Complete remission (CR)
Time frame: Measured up to 2 years after the last participant is enrolled
Partial remission (PR)
Time frame: Measured up to 2 years after the last participant is enrolled
CR with limited count recovery (CRL)
Time frame: Measured up to 2 years after the last participant is enrolled
CR with partial hematologic recovery (CRh)
Time frame: Measured up to 2 years after the last participant is enrolled
Progression-free survival (PFS)
Time frame: Measured up to 4 years after the last participant is enrolled
Treatment-related mortality (TRM)
Time frame: Measured up to 2 years after the last participant is enrolled
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 Oct 2026.
- Individual participant dataSharing planned: No
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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.
Bo Cai, MD principal investigator
Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital
1 site 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 (AZA) or Decitabine (DAC) and GPBMC infusion in people with Myelodysplastic Syndromes, run by Beijing 302 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 10 inclusion and 8 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 9 Oct 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 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?
Contact Bo Cai, MD (+861066947168, caibo2008@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (OS) (time frame: Measured up to 4 years after the last participant is enrolled). The study lists 2 primary outcomes in all. 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 Oct 2026.