Older PatientsPhase 2/3NCT07052422
VEN+DAC+Bu2Flu4 vs Bu2Flu5 Conditioning Regimen for Elderly Myeloid Malignancies Undergoing Allo-HSCT
A Phase 2/3 study testing Venetoclax (VEN), Decitabine (DAC) and Busulfan (Bu) in people with Older Patients and Myeloid Malignancies, run by Nanfang Hospital, Southern Medical University.
Trial summary
The purpose of this study is to compare the efficacy and safety of venetoclax+decitabine+busulfan+fludarabine (VEN+DAC+Bu2Flu4) regimen with busulfan+fludarabine (Bu2Flu5) regimen in older patients with myeloid malignancies undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Read more about this study
- Who is it for?
- Ages 60 to 75 years
- Conditions the study is for
- Older Patients, Myeloid Malignancies, Conditioning
- What is being tested?
- Venetoclax (VEN), Decitabine (DAC) and Busulfan (Bu)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- No sites listed yet
- Study dates
- 15 Jul 2025to 31 Dec 2029
- Phase
- Phase 2/3
- 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 60 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 60-75 years
- Acute myeloid leukaemia in first complete remission or myelodysplastic syndrome
- Willing to undergo the first allo-HSCT
- Eastern Cooperative Oncology Group performance status of 0-2
Who cannot join
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (investigators' decision)
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Venetoclax+Decitabine+Busulfan+Fludarabine (VEN+DAC+Bu2Flu4) vs Busulfan+Fludarabine Conditioning Regimen (Bu2Flu5 ) for Older Patients With Myeloid Malignancies Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
The purpose of this study is to compare the efficacy and safety of venetoclax+decitabine+busulfan+fludarabine (VEN+DAC+Bu2Flu4) regimen with busulfan+fludarabine (Bu2Flu5) regimen in older patients with myeloid malignancies undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Read the detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potent curative approach for myeloid malignancies, but outcomes post-transplantation of older population were unsatisfactory. The conditioning regimen is an essential factor affecting outcomes post-transplantation. Currently, the optimal conditioning for older patients with myeloid malignancies remains unclear. Myeloablative conditioning (MAC) regimens like busulfan plus cyclophosphamide, and busulfan plus fludarabine (Bu4Flu4-5) have low relapse rates, but high non-relapse mortality (NRM) is observed in older patients with myeloid malignancies. The development of reduced-intensity conditioning (RIC) regimens such as Bu2Flu5 has decreased NRM and enhanced the feasibility of allo-HSCT in older patients with myeloid malignancies, but it appears to have higher relapse rate. Neither conventional MAC nor RIC regimens benefit older patients with myeloid malignancies. In recent years, some studies reported that the introduction of venetoclax (VEN) or decitabine (DAC) to MAC reduced relapse without increasing NRM in younger patients with high-risk myeloid malignancies. However, whether VEN and DAC combined with RIC regimen reduce relapse without increasing NRM, then improve survival in older patients with myeloid malignancies is unclear. Therefore, we conducted a randomized controlled study to compare the efficacy and safety of VEN+DAC+Bu2Flu4 with Bu2Flu5 in older patients with myeloid malignancies undergoing allo-HSCT.
- Conditions
- Older Patients, Myeloid Malignancies, Conditioning, Hematopoietic Stem Cell Transplantation (HSCT)
- Being tested
- Venetoclax (VEN), Decitabine (DAC), Busulfan (Bu), Fludarabine (Flu)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Venetoclax (VEN)Drug
Venetoclax (VEN) was administered at 400mg/day on days -10 to -4.
Decitabine (DAC)Drug
Decitabine (DAC) was administered at 20mg/m2/day on days
-10 to -8.
Busulfan (Bu)Drug
Busulfan (Bu) was administered at 3.2 mg/kg/day on days
-5 to -4.
Fludarabine (Flu)Drug
Fludarabine (Flu) was administered at 30mg/m2/day on days -5 to -2.
Fludarabine (Flu)Drug
Fludarabine (Flu) was administered at 30mg/m2/day on days -6 to -2.
| Study group | Role | What they receive |
|---|---|---|
| VEN+DAC+Bu2Flu4 | Experimental | Venetoclax+Decitabine+Busulfan+Fludarabine Receives: Drug: Venetoclax (VEN); Drug: Decitabine (DAC); Drug: Busulfan (Bu); Drug: Fludarabine (Flu) |
| Bu2Flu5 | Active comparator | Busulfan+Fludarabine Receives: Drug: Busulfan (Bu); Drug: Fludarabine (Flu) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease-free survival (DFS) rate
Will calculate time from random assignment until disease progression or relapse or death from any cause
Measured: 2 year
Secondary outcomes (3)
Overall survival (OS) rate
Will calculate time from random assignment until death from any cause.
Measured: 2 year
Relapse incidence
Will calculate time from random assignment until relapse or disease progression.
Measured: 2 year
Non-relapse mortality (NRM) incidence
Defined as death from any cause not subsequent to relapse or disease progression.
Measured: 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2025
- Study start15 Jul 2025 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Older PatientsStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 160 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Qifa Liu, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07052422
- Sponsor's protocol ID
- NFEC-2025-260
- Status last verified
- Jul 2025
- First submitted
- 22 Jun 2025
- Last updated
- 4 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ling Y, Xuan L, Xu N, Huang F, Fan Z, Guo Z, Xu X, Liu H, Lin R, Yu S, Zhang H, Jin H, Wu M, Liu C, Liang X, Ou R, Zhang Y, Liu X, Qu H, Zhai X, Sun J, Zhao Y, Liu Q. Busulfan Plus Fludarabine Compared With Busulfan Plus Cyclophosphamide for AML Undergoing HLA-Haploidentical Hematopoietic Cell Transplantation: A Multicenter Randomized Phase III Trial. J Clin Oncol. 2023 Oct 10;41(29):4632-4642. doi: 10.1200/JCO.23.00101. Epub 2023 Jun 19.
PubMed 37335960Rambaldi A, Grassi A, Masciulli A, Boschini C, Mico MC, Busca A, Bruno B, Cavattoni I, Santarone S, Raimondi R, Montanari M, Milone G, Chiusolo P, Pastore D, Guidi S, Patriarca F, Risitano AM, Saporiti G, Pini M, Terruzzi E, Arcese W, Marotta G, Carella AM, Nagler A, Russo D, Corradini P, Alessandrino EP, Torelli GF, Scime R, Mordini N, Oldani E, Marfisi RM, Bacigalupo A, Bosi A. Busulfan plus cyclophosphamide versus busulfan plus fludarabine as a preparative regimen for allogeneic haemopoietic stem-cell transplantation in patients with acute myeloid leukaemia: an open-label, multicentre, randomised, phase 3 trial. Lancet Oncol. 2015 Nov;16(15):1525-1536. doi: 10.1016/S1470-2045(15)00200-4. Epub 2015 Sep 28.
PubMed 26429297Devine SM, Owzar K, Blum W, Mulkey F, Stone RM, Hsu JW, Champlin RE, Chen YB, Vij R, Slack J, Soiffer RJ, Larson RA, Shea TC, Hars V, Sibley AB, Giralt S, Carter S, Horowitz MM, Linker C, Alyea EP. Phase II Study of Allogeneic Transplantation for Older Patients With Acute Myeloid Leukemia in First Complete Remission Using a Reduced-Intensity Conditioning Regimen: Results From Cancer and Leukemia Group B 100103 (Alliance for Clinical Trials in Oncology)/Blood and Marrow Transplant Clinical Trial Network 0502. J Clin Oncol. 2015 Dec 10;33(35):4167-75. doi: 10.1200/JCO.2015.62.7273. Epub 2015 Nov 2.
PubMed 26527780Xuan L, Dai M, Jiang E, Wang Y, Huang F, Fan Z, Xu N, Nie D, Liang X, Chen H, Ye J, Shi P, Liu H, Jin H, Lin R, Yan C, Zhang Y, Sun J, Han M, Liu Q. The effect of granulocyte-colony stimulating factor, decitabine, and busulfan-cyclophosphamide versus busulfan-cyclophosphamide conditioning on relapse in patients with myelodysplastic syndrome or secondary acute myeloid leukaemia evolving from myelodysplastic syndrome undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2023 Mar;10(3):e178-e190. doi: 10.1016/S2352-3026(22)00375-1. Epub 2023 Jan 23.
PubMed 36702138Zheng X, Gao H, Lu N, Wang M, Zhang H, Zheng Y, Shen B, Cao Y, Chen X, Zhai W, Wei J, Yang D, Zhang R, Pang A, Feng S, Jiang E, Han M. Efficacy of venetoclax combined with decitabine conditioning regimen for allogeneic hematopoietic stem cell transplantation in high-risk and elderly patients with myeloid neoplasms. Ann Hematol. 2023 Dec;102(12):3603-3611. doi: 10.1007/s00277-023-05500-2. Epub 2023 Oct 25.
PubMed 37878011
Study at a glance
From the registry record, last updated by the sponsor on 4 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Venetoclax+Decitabine+Busulfan+Fludarabine (VEN+DAC+Bu2Flu4) vs Busulfan+Fludarabine Conditioning Regimen (Bu2Flu5 ) for Older Patients With Myeloid Malignancies Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
- ClinicalTrials.gov ID
- NCT07052422
- Acronym / sponsor ID
- NFEC-2025-260
- Conditions
- Older Patients, Myeloid Malignancies, Conditioning, Hematopoietic Stem Cell Transplantation (HSCT)
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Study start
- 15 Jul 2025 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 4 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.
The purpose of this study is to compare the efficacy and safety of venetoclax+decitabine+busulfan+fludarabine (VEN+DAC+Bu2Flu4) regimen with busulfan+fludarabine (Bu2Flu5) regimen in older patients with myeloid malignancies undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potent curative approach for myeloid malignancies, but outcomes post-transplantation of older population were unsatisfactory. The conditioning regimen is an essential factor affecting outcomes post-transplantation. Currently, the optimal conditioning for older patients with myeloid malignancies remains unclear. Myeloablative conditioning (MAC) regimens like busulfan plus cyclophosphamide, and busulfan plus fludarabine (Bu4Flu4-5) have low relapse rates, but high non-relapse mortality (NRM) is observed in older patients with myeloid malignancies. The development of reduced-intensity conditioning (RIC) regimens such as Bu2Flu5 has decreased NRM and enhanced the feasibility of allo-HSCT in older patients with myeloid malignancies, but it appears to have higher relapse rate. Neither conventional MAC nor RIC regimens benefit older patients with myeloid malignancies. In recent years, some studies reported that the introduction of venetoclax (VEN) or decitabine (DAC) to MAC reduced relapse without increasing NRM in younger patients with high-risk myeloid malignancies. However, whether VEN and DAC combined with RIC regimen reduce relapse without increasing NRM, then improve survival in older patients with myeloid malignancies is unclear. Therefore, we conducted a randomized controlled study to compare the efficacy and safety of VEN+DAC+Bu2Flu4 with Bu2Flu5 in older patients with myeloid malignancies undergoing allo-HSCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VEN+DAC+Bu2Flu4 | Experimental | Drug: Venetoclax (VEN), Drug: Decitabine (DAC), Drug: Busulfan (Bu), Drug: Fludarabine (Flu)Venetoclax+Decitabine+Busulfan+Fludarabine |
| Bu2Flu5 | Active comparator | Drug: Busulfan (Bu), Drug: Fludarabine (Flu)Busulfan+Fludarabine |
Who may qualify
- Ages
- 60 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 60-75 years
- Acute myeloid leukaemia in first complete remission or myelodysplastic syndrome
- Willing to undergo the first allo-HSCT
- Eastern Cooperative Oncology Group performance status of 0-2
Key exclusion criteria (2 listed)
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (investigators' decision)
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 (VEN)Venetoclax (VEN) was administered at 400mg/day on days -10 to -4. | Drug | VEN+DAC+Bu2Flu4 |
| Decitabine (DAC)Decitabine (DAC) was administered at 20mg/m2/day on days -10 to -8. | Drug | VEN+DAC+Bu2Flu4 |
| Busulfan (Bu)Busulfan (Bu) was administered at 3.2 mg/kg/day on days -5 to -4. | Drug | Bu2Flu5, VEN+DAC+Bu2Flu4 |
| Fludarabine (Flu)Fludarabine (Flu) was administered at 30mg/m2/day on days -5 to -2. | Drug | VEN+DAC+Bu2Flu4 |
| Fludarabine (Flu)Fludarabine (Flu) was administered at 30mg/m2/day on days -6 to -2. | Drug | Bu2Flu5 |
Primary outcome
Disease-free survival (DFS) rate
Time frame: 2 year
Will calculate time from random assignment until disease progression or relapse or death from any cause
3 secondary outcomes
Overall survival (OS) rate
Time frame: 2 year
Relapse incidence
Time frame: 2 year
Non-relapse mortality (NRM) incidence
Time frame: 2 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 4 Jul 2025.
- Background: PubMed 37335960Ling Y, Xuan L, Xu N, Huang F, Fan Z, Guo Z, Xu X, Liu H, Lin R, Yu S, Zhang H, Jin H, Wu M, Liu C, Liang X, Ou R, Zhang Y, Liu X, Qu H, Zhai X, Sun J, Zhao Y, Liu Q. Busulfan Plus Fludarabine Compared With Busulfan Plus Cyclophosphamide for AML Undergoing HLA-Haploidentical Hematopoietic Cell Transplantation: A Multicenter Randomized Phase III Trial. J Clin Oncol. 2023 Oct 10;41(29):4632-4642. doi: 10.1200/JCO.23.00101. Epub 2023 Jun 19.
- Background: PubMed 26429297Rambaldi A, Grassi A, Masciulli A, Boschini C, Mico MC, Busca A, Bruno B, Cavattoni I, Santarone S, Raimondi R, Montanari M, Milone G, Chiusolo P, Pastore D, Guidi S, Patriarca F, Risitano AM, Saporiti G, Pini M, Terruzzi E, Arcese W, Marotta G, Carella AM, Nagler A, Russo D, Corradini P, Alessandrino EP, Torelli GF, Scime R, Mordini N, Oldani E, Marfisi RM, Bacigalupo A, Bosi A. Busulfan plus cyclophosphamide versus busulfan plus fludarabine as a preparative regimen for allogeneic haemopoietic stem-cell transplantation in patients with acute myeloid leukaemia: an open-label, multicentre, randomised, phase 3 trial. Lancet Oncol. 2015 Nov;16(15):1525-1536. doi: 10.1016/S1470-2045(15)00200-4. Epub 2015 Sep 28.
- Background: PubMed 26527780Devine SM, Owzar K, Blum W, Mulkey F, Stone RM, Hsu JW, Champlin RE, Chen YB, Vij R, Slack J, Soiffer RJ, Larson RA, Shea TC, Hars V, Sibley AB, Giralt S, Carter S, Horowitz MM, Linker C, Alyea EP. Phase II Study of Allogeneic Transplantation for Older Patients With Acute Myeloid Leukemia in First Complete Remission Using a Reduced-Intensity Conditioning Regimen: Results From Cancer and Leukemia Group B 100103 (Alliance for Clinical Trials in Oncology)/Blood and Marrow Transplant Clinical Trial Network 0502. J Clin Oncol. 2015 Dec 10;33(35):4167-75. doi: 10.1200/JCO.2015.62.7273. Epub 2015 Nov 2.
- Background: PubMed 36702138Xuan L, Dai M, Jiang E, Wang Y, Huang F, Fan Z, Xu N, Nie D, Liang X, Chen H, Ye J, Shi P, Liu H, Jin H, Lin R, Yan C, Zhang Y, Sun J, Han M, Liu Q. The effect of granulocyte-colony stimulating factor, decitabine, and busulfan-cyclophosphamide versus busulfan-cyclophosphamide conditioning on relapse in patients with myelodysplastic syndrome or secondary acute myeloid leukaemia evolving from myelodysplastic syndrome undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2023 Mar;10(3):e178-e190. doi: 10.1016/S2352-3026(22)00375-1. Epub 2023 Jan 23.
- Background: PubMed 37878011Zheng X, Gao H, Lu N, Wang M, Zhang H, Zheng Y, Shen B, Cao Y, Chen X, Zhai W, Wei J, Yang D, Zhang R, Pang A, Feng S, Jiang E, Han M. Efficacy of venetoclax combined with decitabine conditioning regimen for allogeneic hematopoietic stem cell transplantation in high-risk and elderly patients with myeloid neoplasms. Ann Hematol. 2023 Dec;102(12):3603-3611. doi: 10.1007/s00277-023-05500-2. Epub 2023 Oct 25.
- 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.
Qifa Liu principal investigator
Nanfang Hospital, Southern Medical University
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Venetoclax (VEN), Decitabine (DAC) and Busulfan (Bu) in people with Older Patients and Myeloid Malignancies, run by Nanfang Hospital, Southern Medical University.
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 4 inclusion and 2 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: opening soon, as of the sponsor's last update on 4 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 Qifa Liu (+86-020-62787883, liuqifa628@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease-free survival (DFS) rate (time frame: 2 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07052422, which also lists 5 linked publications.
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 4 Jul 2025.