Allogeneic Hematopoietic Stem Cell TransplantationPhase 2NCT05809167
Venetoclax+Azacytidine+Modified BUCY Conditioning Regimen for Acute Lymphoblastic Leukemia Undergoing Allo-HSCT
A Phase 2 study testing VEN+AZA+Modified BUCY in people with Allogeneic Hematopoietic Stem Cell Transplantation and Acute Lymphoblastic 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 or relapsed/refractory acute lymphoblastic leukemia (ALL) undergoing allogeneic… 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 Lymphoblastic Leukemia
- What is being tested?
- VEN+AZA+Modified BUCY
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 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 14 to 65 years;
- Diagnosis of high-risk or relapsed/refractory acute lymphoblastic leukemia at the time of transplant.
- 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 <14 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 (VEN)+Azacytidine (AZA) Followed by Modified BUCY Conditioning Regimen for High Risk or Refractory/Relapsed Acute Lymphoblastic Leukemia 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 or relapsed/refractory acute lymphoblastic leukemia (ALL) 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 acute lymphoblastic leukemia. 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 ALL 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. Multiple case reports the efficacy of Venetoclax-based regimens in patients with ALL is encouraging. The objective of this study is to evaluate the feasibility, safety and efficacy of VEN-AZA bridging allogeneic hematopoietic stem cell transplantation in the treatment of high-risk ALL.
- Being tested
- VEN+AZA+Modified BUCY
- Keywords
- venetoclax, azacytidine, hematopoietic stem cell transplantation, acute lymphoblastic leukemia
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
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 regimen related toxicity as assessed by CTCAE v4.0
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 posted12 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
- 55 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
- NCT05809167
- Sponsor's protocol ID
- VA+mBUCY-02
- Status last verified
- Feb 2023
- First submitted
- 27 Feb 2023
- Last updated
- 12 Apr 2023
- 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 12 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Venetoclax (VEN)+Azacytidine (AZA) Followed by Modified BUCY Conditioning Regimen for High Risk or Refractory/Relapsed Acute Lymphoblastic Leukemia Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
- ClinicalTrials.gov ID
- NCT05809167
- Acronym / sponsor ID
- VA+mBUCY-02
- Keywords
- venetoclax, azacytidine, hematopoietic stem cell transplantation, acute lymphoblastic leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 55 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
- 12 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 or relapsed/refractory acute lymphoblastic leukemia (ALL) 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 acute lymphoblastic leukemia. 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 ALL 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. Multiple case reports the efficacy of Venetoclax-based regimens in patients with ALL is encouraging. The objective of this study is to evaluate the feasibility, safety and efficacy of VEN-AZA bridging allogeneic hematopoietic stem cell transplantation in the treatment of high-risk ALL.
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 14 to 65 years;
- Diagnosis of high-risk or relapsed/refractory acute lymphoblastic leukemia at the time of transplant.
- 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 <14 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 12 Apr 2023.
- 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. 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 Lymphoblastic 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 12 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 (+86-512-6778185, 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 12 Apr 2023.