Myelodysplastic NeoplasmsPhase 2NCT07047183
Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes
A Phase 2 study testing VEN (Venetoclax) in people with Myelodysplastic Neoplasms and Transplantation, run by Yehui Tan at 1 site in China.
Trial summary
A Single-Arm, Prospective Clinical Study of Venetoclax Combined with Azacitidine Followed by Bridging Transplantation in Patients with High-Risk Myelodysplastic Neoplasms with Increased Blasts 2 (MDS-IB2) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Neoplasms, Transplantation
- What is being tested?
- VEN (Venetoclax)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2025to 30 Jun 2029
- 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
- Newly diagnosed MDS confirmed by morphological and immunophenotypic analysis of bone marrow；
- Age ≥18 years, any gender；
- Bone marrow blasts ≥10%；
- IPSS-R score >4.5；
- ECOG performance status 0-2；
- Scheduled for allogeneic hematopoietic stem cell transplantation (allo-HSCT)；
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- Adequate major organ function：
- Cardiac: LVEF ≥50%
- Hepatic: Bilirubin ≤1.5×ULN
- AST/ALT ≤2.5×ULN
- Renal: Creatinine clearance ≥60 mL/min；
- Written informed consent provided by the patient or legally authorized representative.
Who cannot join
- Extramedullary disease involvement；
- Hypersensitivity to any study drugs；
- Clinically significant hepatic/renal dysfunction exceeding inclusion thresholds；
- Severe cardiac disease, including congestive heart failure, myocardial infarction, and cardiac insufficiency;
- Concurrent malignant tumors of other organs, which can be enrolled if previously cured;
- Active tuberculosis or HIV infection;
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- Concomitant hematologic disorders;
- Pregnancy or lactation;
- Inability to comply with protocol requirements;
- Concurrently participating in other clinical studies.
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
The First Hospital of Jilin University
Changchun, Jilin, 130000
About this study
Official title: A Single-Arm, Prospective Clinical Study of Venetoclax Combined With Azacitidine Followed by Bridging Transplantation in Patients With High-Risk Myelodysplastic Neoplasms With Increased Blasts 2 (MDS-IB2)
A Single-Arm, Prospective Clinical Study of Venetoclax Combined with Azacitidine Followed by Bridging Transplantation in Patients with High-Risk Myelodysplastic Neoplasms with Increased Blasts 2 (MDS-IB2)
Read the detailed description
This is a single-center, single-arm, prospective study investigating pre-transplant bridging therapy in patients aged ≥18 years with higher-risk MDS-IB2. The study plans to enroll 46 eligible patients. Participants will receive Venetoclax combined with Azacitidine, administered in 28-day cycles for 1 to 2 cycles. Treatment response will be assessed according to the IWG 2023 HR-MDS response criteria.Patients achieving modified Composite Complete Remission (mCRc: CR or CR-equivalent + CRL + CRh) after Cycle 1 will proceed directly to transplantation. Patients not achieving mCRc will receive a second cycle of therapy. All patients will undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) within 3 months after completing Cycle 2, regardless of mCRc status. Patients unable to proceed to transplantation will receive standard institutional care and undergo follow-up.
- Conditions
- Myelodysplastic Neoplasms, Transplantation
- Being tested
- VEN (Venetoclax)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VEN (Venetoclax)Drug
Also called Azacitidine
Enrolled patients will receive: Venetoclax: 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Days 3-14. Azacitidine: 75 mg/m² on Days 1-7. Cycle duration: 28 days Total cycles: 1 to 2 cycles
| Study group | Role | What they receive |
|---|---|---|
| venetoclax + Azacitidine | Experimental | Not described Receives: Drug: VEN (Venetoclax) |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-Year Relapse-Free Survival(2-RFS)
Defined as the time from enrollment in this study to the occurrence of relapse. For patients without relapse, the time is calculated until death from any cause or the time of the last follow-up
Measured: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.
Secondary outcomes (3)
Overall Survival (OS)
Defined as the time from enrollment in this study to death from any cause. If the exact date of death is unknown, the time of death is defined as the last contact date. If the patient is still alive, the last observed time will be used, and the patient's OS will be considered for analysis.
Measured: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 48 months.
Cumulative Relapse Rate (CIR)
Defined as the proportion of patients who experience disease relapse from the time of enrollment in this study.
Measured: rom date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.
Composite Complete Response Rate（mCRc）
Complete Remission (CR) or CR-equivalent+ CR with partial hematologic recovery (CRL) + CR with limited hematologic recovery (CRh)
Measured: Through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2025
- Study start1 Jul 2025
- Primary completion30 Jun 2029 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic NeoplasmsStudies 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
- 46 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yehui Tan
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Yehui Tan, The First Hospital of Jilin University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07047183
- Sponsor's protocol ID
- 2025-HS-061
- Status last verified
- Aug 2025
- First submitted
- 24 Jun 2025
- Last updated
- 8 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm, Prospective Clinical Study of Venetoclax Combined With Azacitidine Followed by Bridging Transplantation in Patients With High-Risk Myelodysplastic Neoplasms With Increased Blasts 2 (MDS-IB2)
- ClinicalTrials.gov ID
- NCT07047183
- Acronym / sponsor ID
- 2025-HS-061
- Conditions
- Myelodysplastic Neoplasms, Transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 46 participants (estimated)
- Lead sponsor
- Yehui TanUniversity, hospital or other
- Study start
- 1 Jul 2025 (actual)
- Primary completion
- 30 Jun 2029 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 2 Jul 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.
A Single-Arm, Prospective Clinical Study of Venetoclax Combined with Azacitidine Followed by Bridging Transplantation in Patients with High-Risk Myelodysplastic Neoplasms with Increased Blasts 2 (MDS-IB2)
Detailed description
This is a single-center, single-arm, prospective study investigating pre-transplant bridging therapy in patients aged ≥18 years with higher-risk MDS-IB2. The study plans to enroll 46 eligible patients. Participants will receive Venetoclax combined with Azacitidine, administered in 28-day cycles for 1 to 2 cycles. Treatment response will be assessed according to the IWG 2023 HR-MDS response criteria.Patients achieving modified Composite Complete Remission (mCRc: CR or CR-equivalent + CRL + CRh) after Cycle 1 will proceed directly to transplantation. Patients not achieving mCRc will receive a second cycle of therapy. All patients will undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) within 3 months after completing Cycle 2, regardless of mCRc status. Patients unable to proceed to transplantation will receive standard institutional care and undergo follow-up.
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 | Experimental | Drug: VEN (Venetoclax) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Newly diagnosed MDS confirmed by morphological and immunophenotypic analysis of bone marrow；
- Age ≥18 years, any gender；
- Bone marrow blasts ≥10%；
- IPSS-R score >4.5；
- ECOG performance status 0-2；
Key exclusion criteria (10 listed)
- Extramedullary disease involvement；
- Hypersensitivity to any study drugs；
- Clinically significant hepatic/renal dysfunction exceeding inclusion thresholds；
- Severe cardiac disease, including congestive heart failure, myocardial infarction, and cardiac insufficiency;
- Concurrent malignant tumors of other organs, which can be enrolled if previously cured;
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 |
|---|---|---|
| VEN (Venetoclax)Also: AzacitidineEnrolled patients will receive: Venetoclax: 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Days 3-14. Azacitidine: 75 mg/m² on Days 1-7. Cycle duration: 28 days Total cycles: 1 to 2 cycles | Drug | venetoclax + Azacitidine |
Primary outcome
2-Year Relapse-Free Survival(2-RFS)
Time frame: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.
Defined as the time from enrollment in this study to the occurrence of relapse. For patients without relapse, the time is calculated until death from any cause or the time of the last follow-up
3 secondary outcomes
Overall Survival (OS)
Time frame: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 48 months.
Cumulative Relapse Rate (CIR)
Time frame: rom date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.
Composite Complete Response Rate（mCRc）
Time frame: Through study completion, an average of 1 year
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 8 Aug 2025.
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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.
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 VEN (Venetoclax) in people with Myelodysplastic Neoplasms and Transplantation, run by Yehui Tan 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 12 inclusion and 10 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 8 Aug 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.
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 Yehui Tan (8615948027438, yhtan@jlu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-Year Relapse-Free Survival(2-RFS) (time frame: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 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 8 Aug 2025.