Allogeneic Hematopoietic Stem Cell TransplantationPhase 2NCT05823714
Venetoclax+Azacytidine+Modified BUCY Conditioning Regimen for Acute Myeloid Leukemia and Myelodysplastic Syndromes Undergoing Allo-HSCT
A Phase 2 study testing VEN+AZA+Modified BUCY in people with Allogeneic Hematopoietic Stem Cell Transplantation and Acute Myeloid Leukemia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
The purpose of this prospective, open-label, single-center study is to evaluate the efficacy and safety of VEN-AZA (venetoclax and azacytidine) followed by modified BUCY (busulfan and cyclophosphamide) as conditioning regimen for high-risk myelodysplastic syndrome (MDS) and high-risk or relapsed/refractory acute… Read more about this study
- Who is it for?
- Ages 8 to 65 years
- Conditions the study is for
- Allogeneic Hematopoietic Stem Cell Transplantation, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- What is being tested?
- VEN+AZA+Modified BUCY
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteChina
- Study dates
- 20 Jan 2022to 20 Jan 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 8 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age 8 to 65 years;
- Diseases must be MDS and AML; high-risk MDS: int-2 risk, high risk by the IPSS; IPSS-R int-risk (> 3.5 points), high risk, very high risk; high risk,very high risk by the WPSS; high-risk or relapsed/refractory AML: (1) age≥60 years; (2) High white blood cell count at first diagnosis (WBC≥100*10^9/L); (3) secondary AML (previous history of MDS, myeloproliferative disease, or treatment-related AML, etc.); (4) Complicated with extramedullary leukemia, such as central nervous system leukemia, granulocytic sarcoma, hepatosenomegaly; （5）high risk factors and relapsed/refractory AML（reference 2022-AML-ELN guideline）（6）not in remission or ≥CR2 before transplantation;
- Must need a bone marrow transplant;
- Must have the ability to observe the efficacy and events;
- Patient must have ability to understand and willingness to provide written informed consent prior to participation in the study and any related procedures being performed.
Who cannot join
- Age <8 or >65 years;
- Uncontrolled bacterial, viral, fungal, or other infection before conditioning regimen;
- Pregnant or lactating females;
- Current participation in another clinical trial;
- Contra-indication to one of the drug of the regimen;
- Any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart, kidney, liver or other organ system that may place the patient at undue risk to undergo the agents included in the conditioning regimen.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
Xiaowen Tang, PhD, 67781525, xwtang1020@163.com
About this study
Official title: Venetoclax + Azacytidine Followed by Modified BUCY Conditioning Regimen for High-risk Myelodysplastic Syndromes (MDS) and High-risk or Relapsed/Refractory Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
The purpose of this prospective, open-label, single-center study is to evaluate the efficacy and safety of VEN-AZA (venetoclax and azacytidine) followed by modified BUCY (busulfan and cyclophosphamide) as conditioning regimen for high-risk myelodysplastic syndrome (MDS) and high-risk or relapsed/refractory acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Read the detailed description
Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is the only potentially curative therapy for patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). However, relapse remains a leading cause for treatment failure after hematopoietic cell transplantation (HCT) in patients, optimization of conditioning regimen can improve prognosis and decrease relapse. Abnormal gene methylation is common in AML and MDS patients. Azacytidine is a DNA methylation transferase inhibitor that can re-express tumor suppressor genes in leukemia cells. Venetoclax is a selective BCL-2 inhibitor, which has antitumor activity against a variety of hematological malignancies. The combination of the two drugs show a synergistic anti-tumor effect. The objective of this study is to evaluate the safety and efficacy of VEN-AZA regimen followed by Allo-HSCT in the treatment of high-risk MDS and high-risk or relapsed/refractory AML.
- Conditions
- Allogeneic Hematopoietic Stem Cell Transplantation, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Being tested
- VEN+AZA+Modified BUCY
- Keywords
- venetoclax, azacytidine, hematopoietic stem cell transplantation, acute myeloid leukemia, myelodysplastic syndrome
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VEN+AZA+Modified BUCYDrug
Venetoclax: 200mg/day*7days（It should be combined with triazole antifungal drugs).
Azacytidine: 75mg/ m²/day*7days.
| Study group | Role | What they receive |
|---|---|---|
| VEN+AZA+Modified BUCY | Experimental | Not described Receives: Drug: VEN+AZA+Modified BUCY |
What is being measured
The primary outcome is the main question the study is designed to answer.
disease-free survival (DFS)
It is measured from the time from randomization to the first of relapse or death.
Measured: 3 years after transplantation
overall survival (OS)
It is measured from the time of entry into this trial to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive.
Measured: 3 years after transplantation
Secondary outcomes (4)
veno-occlusive disease (VOD)
incidence of veno-occlusive disease (VOD) events (refer to modified Seattle Criteria of VOD)
Measured: 3 years after transplantation
graft-versus-host disease (GvHD)
incidence and severity of acute (aGvHD) and chronic graft-versus-host disease (cGvHD) (aGvHD refer to Glucksberg Criteria and cGvHD refer to the National Institutes of Health Consensus)
Measured: 3 years after transplantation
transplant related mortality (TRM)
cumulative incidence of transplant related mortality
Measured: 3 years after transplantation
Regimen related toxicity
Number of participants with conditioning regimen related toxicity
Measured: 3 years after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2023
- Study start20 Jan 2022
- Primary completion20 Jan 2025 (estimated)
- Study completion20 Jan 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Allogeneic Hematopoietic Stem Cell TransplantationStudies 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
- 70 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Depei Wu Wu, MD, Study chair, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05823714
- Sponsor's protocol ID
- VA+mBUCY-01
- Status last verified
- Feb 2023
- First submitted
- 27 Feb 2023
- Last updated
- 21 Apr 2023
- 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 21 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Venetoclax + Azacytidine Followed by Modified BUCY Conditioning Regimen for High-risk Myelodysplastic Syndromes (MDS) and High-risk or Relapsed/Refractory Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
- ClinicalTrials.gov ID
- NCT05823714
- Acronym / sponsor ID
- VA+mBUCY-01
- Conditions
- Allogeneic Hematopoietic Stem Cell Transplantation, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Keywords
- venetoclax, azacytidine, hematopoietic stem cell transplantation, acute myeloid leukemia, myelodysplastic syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 20 Jan 2022 (actual)
- Primary completion
- 20 Jan 2025 (estimated)
- Study completion
- 20 Jan 2026 (estimated)
- First posted
- 21 Apr 2023 (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 prospective, open-label, single-center study is to evaluate the efficacy and safety of VEN-AZA (venetoclax and azacytidine) followed by modified BUCY (busulfan and cyclophosphamide) as conditioning regimen for high-risk myelodysplastic syndrome (MDS) and high-risk or relapsed/refractory acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Detailed description
Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is the only potentially curative therapy for patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). However, relapse remains a leading cause for treatment failure after hematopoietic cell transplantation (HCT) in patients, optimization of conditioning regimen can improve prognosis and decrease relapse. Abnormal gene methylation is common in AML and MDS patients. Azacytidine is a DNA methylation transferase inhibitor that can re-express tumor suppressor genes in leukemia cells. Venetoclax is a selective BCL-2 inhibitor, which has antitumor activity against a variety of hematological malignancies. The combination of the two drugs show a synergistic anti-tumor effect. The objective of this study is to evaluate the safety and efficacy of VEN-AZA regimen followed by Allo-HSCT in the treatment of high-risk MDS and high-risk or relapsed/refractory AML.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VEN+AZA+Modified BUCY | Experimental | Drug: VEN+AZA+Modified BUCY |
Who may qualify
- Ages
- 8 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Age 8 to 65 years;
- Diseases must be MDS and AML; high-risk MDS: int-2 risk, high risk by the IPSS; IPSS-R int-risk (> 3.5 points), high risk, very high risk; high risk,very high risk by the WPSS; high-risk or relapsed/refractory AML: (1) age≥60 years; (2) High white blood cell count at first diagnosis (WBC≥100*10^9/L); (3) secondary AML (previous history of MDS, myeloproliferative disease, or treatment-related AML, etc.); (4) Complicated with extramedullary leukemia, such as central nervous system leukemia, granulocytic sarcoma, hepatosenomegaly; （5）high risk factors and relapsed/refractory AML（reference 2022-AML-ELN guideline）（6）not in remission or ≥CR2 before transplantation;
- Must need a bone marrow transplant;
- Must have the ability to observe the efficacy and events;
- Patient must have ability to understand and willingness to provide written informed consent prior to participation in the study and any related procedures being performed.
Key exclusion criteria (6 listed)
- Age <8 or >65 years;
- Uncontrolled bacterial, viral, fungal, or other infection before conditioning regimen;
- Pregnant or lactating females;
- Current participation in another clinical trial;
- Contra-indication to one of the drug of the regimen;
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+AZA+Modified BUCYVenetoclax: 200mg/day*7days（It should be combined with triazole antifungal drugs). Azacytidine: 75mg/ m²/day*7days. | Drug | VEN+AZA+Modified BUCY |
Primary outcome
disease-free survival (DFS)
Time frame: 3 years after transplantation
It is measured from the time from randomization to the first of relapse or death.
overall survival (OS)
Time frame: 3 years after transplantation
It is measured from the time of entry into this trial to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive.
4 secondary outcomes
veno-occlusive disease (VOD)
Time frame: 3 years after transplantation
graft-versus-host disease (GvHD)
Time frame: 3 years after transplantation
transplant related mortality (TRM)
Time frame: 3 years after transplantation
Regimen related toxicity
Time frame: 3 years after transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2023.
Related trials for Allogeneic Hematopoietic Stem Cell Transplantation
Active, not recruitingPhase 2
De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
Graft-versus-host Disease
Not yet recruitingObservational study
Systematic Intraoperative Cell Salvage in On-Pump Cardiac Surgery Within a Mature Patient Blood Management Programme
Cardiac Surgical Procedures, Blood Transfusion
RecruitingObservational study
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
Graft-Versus-Host Disease, Graft-versus-leukemia
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingObservational study
Natural History Study to Determine Drug Metabolism Phenotype and Appropriate Germline Source DNA in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant
Leukemia, Lymphoma
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.
Depei Wu Wu, MD study chair
The First Affiliated Hospital of Soochow University
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+AZA+Modified BUCY in people with Allogeneic Hematopoietic Stem Cell Transplantation and Acute Myeloid Leukemia, run by The First Affiliated Hospital of Soochow University 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 5 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 21 Apr 2023. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiaowen Tang, MD (+86-512-67781851, xwtang1020@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) (time frame: 3 years after transplantation). 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 21 Apr 2023.