Hematopoietic Stem Cell TransplantationPhase 2NCT05528354
Venetoclax and Decitabin Based Conditioning Regimen Followed With Post-HSCT Decitabin Maintenance Therapy in TP53 Mutant AML/MDS Patients
A Phase 2 study testing VEN and DEC based conditioning regimen and DEC in people with Hematopoietic Stem Cell Transplantation and TP53, run by Zhejiang University at 4 sites in China.
Trial summary
This study aims to evaluate the effectiveness and safety of Venetoclax and Decitabin based conditioning regimen followed with post-HSCT Decitabin maintenance therapy in TP53 mutant AML/MDS Patients. Read more about this study
- Who is it for?
- Ages 12 to 70 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplantation, TP53
- What is being tested?
- VEN and DEC based conditioning regimen and DEC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 58 participants
- Where
- 4 sitesChina
- Study dates
- 2 Jun 2022to 31 Mar 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 12 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- AML or MDS diagnosed according to 2016 WHO criteria with TP53 mutation before enrollment;
- Aged from 12 to 70 years;
- The Eastern Cooperative Oncology Group (ECOG) performance score of 0-2;
- Creatinine clearance rate ≥ 60 mL/min (according to Cockcroft-Gault formula);
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≥ 3× upper limit of normal range (ULN), total bilirubin ≤ 2×ULN;
- Left ventricular ejection fraction (LVEF) assessed by echocardiography (ECHO) ≥ 45%;
- Life expectancy > 8 weeks;
- Sign the informed consent voluntarily, understand and comply with all trial requirements.
Who cannot join
- Active autoimmune diseases, such as SLE, rheumatoid arthritis, etc.
- Current active cardiovascular disease with clinically significance, such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease determined by the New York Heart Association (NYHA) functional classification, or a history of myocardial infarction within the 6 months prior to screening;
- Other serious medical conditions (e.g., advanced infection) that may limit the patient's participation in the trial;
- Known human immunodeficiency virus (HIV) infection, or drug-uncontrolled chronic infection of hepatitis B virus (HBV-DNA > 1000IU/ml) or hepatitis C virus (anti-HCV positive);
- Pregnant or lactating women;
- Fail to understand, comply with the study protocol or sign the informed consent form.
Trial locations and contacts
4 sites in 1 country.
China4 sites
The First Hospital of Zhejiang Medical Colleage Zhejiang University
Hangzhou, Zhejiang, 310006
The Second Affiliate Hospital of Nanchang University, Nanchang
Nanchang, 330006
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200025
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, 430030
About this study
This study aims to evaluate the effectiveness and safety of Venetoclax and Decitabin based conditioning regimen followed with post-HSCT Decitabin maintenance therapy in TP53 mutant AML/MDS Patients.
Read the detailed description
In acute myeloid leukemia and myelodysplastic syndromes, TP53 gene mutation is a poor prognostic factor and a strong indication for hematopoietic stem cell transplantation. However, because of the high relapse rate of myeloid tumors with TP53 mutation, new comprehensive treatment is urgently needed to improve the efficacy of transplantation. Decitabin(DEC) has shown certain efficacy in primary patients with TP53 mutation, and Venetoclax (VEN) and DEC have synergistic effect. Based on this, we hypothesized that DEC and VEN should be added to the conditioning regimen in TP53 mutant AML/MDS patients in order to eliminate malignant clones with P53 mutation as much as possible, and intermittent DEC maintenance therapy should be used to prevent relapse after post-HSCT hematopoietic reconstruction. We intend to conduct a multicenter, single-arm clinical study to evaluate the efficacy and safety of this protocol.
- Conditions
- Hematopoietic Stem Cell Transplantation, TP53
- Being tested
- VEN and DEC based conditioning regimen, DEC
- Keywords
- hematopoietic stem cell transplantation, TP53, relapse, decitabine
- Medical subject headings
- Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VEN and DEC based conditioning regimenDrug
For human leukocyte antigen (HLA) matched-donor transplantation, the regimen consisted of venetoclax 400 mg once daily on days -12 to -6, decitabine 20 mg/m² once daily on days -12 to -8, busulfan 0.8 mg/kg every 6 h on days -7 to -4, cyclophosphamide 60 mg/kg once daily on days -3 to -2, and semustine 250 mg/m² on day -1.
For haploidentical HCT, the regimen consisted of venetoclax 400 mg once daily on days -14 to -8, decitabine 20 mg/m² once daily on days -14 to -10, cytarabine 2 g/m² once daily on days -9 to -8, busulfan 0.8 mg/kg every 6 h on days -7 to -5, cyclophosphamide 1.8 g/m² once daily on days -4 to -3, and semustine 250 mg/m² on day -2.
Patients aged ≥50 years or with an HCT-CI score ≥3 received reduced-toxicity conditioning (RTC), consisting of venetoclax 400 mg once daily on days -12 to -6, decitabine 20 mg/m² once daily on days -12 to -8, fludarabine 30 mg/m² once daily on days -7 to -3, busulfan 0.8 mg/kg every 6 h on days -7 to -5, and semustine 250 mg/m² on day -2.
DECDrug
In the time window of 60-120 days after transplantation: DEC 5mg/m2/d for 5 consecutive days every 6 to 8 weeks with a total of 6 courses if there is no severe aGVHD (grade 3 or higher) and the donor chimerism rate of bone marrow blood (STR)>95%.
If the MRD turns positive, DLI can be performed.
| Study group | Role | What they receive |
|---|---|---|
| VEN and DEC based conditioning regimen followed with post-HSCT DEC maintenance therapy | Experimental | Not described Receives: Drug: VEN and DEC based conditioning regimen; Drug: DEC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Relapse-free survival (RFS)
Relapse-free survival (RFS) was defined as the time from allo-HCT to disease relapse or progression, or death from any cause, whichever occurred first.
Relapse or progression was defined as the presence of circulating blasts, >5% blasts in the bone marrow, or extramedullary disease after a prior post-HCT complete response, or as persistent morphologic disease in patients who failed to achieve complete response after HCT.
Measured: At Year 1
Secondary outcomes (6)
Overall survival (OS)
Assessment of OS at Year 1
Measured: At Year 1
Cumulative relapse rate
Assessment of cumulative relapse rate at Year 1
Measured: At Year 1
Non-relapse mortality (NRM)
Assessment of NRM at Year 1
Measured: At Year 1
Acute graft-versus-host disease (GVHD)
Acute GVHD incidence
Measured: At Day 100
Chronic graft-versus-host disease (GVHD)
Chronic GVHD incidence
Measured: through study completion, an average of 1 year
Adverse effects
Drug related adverse effects
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 posted6 Sep 2022
- Study start2 Jun 2022
- Primary completion31 Mar 2026
- Study completion31 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Stem Cell TransplantationStudies of the same condition, open and under way.
- More trials run by Zhejiang 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
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, He Huang, Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05528354
- Sponsor's protocol ID
- IIT20220036C-R1
- Status last verified
- Oct 2026
- First submitted
- 28 Aug 2022
- Last updated
- 26 Oct 2026
- Results posted
- 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 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Venetoclax and Decitabin Based Conditioning Regimen Followed With Post-HSCT Decitabin Maintenance Therapy in TP53 Mutant AML/MDS Patients
- ClinicalTrials.gov ID
- NCT05528354
- Acronym / sponsor ID
- IIT20220036C-R1
- Conditions
- Hematopoietic Stem Cell Transplantation, TP53
- Keywords
- hematopoietic stem cell transplantation, TP53, relapse, decitabine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Second Affiliated Hospital of Nanchang University, Tongji Hospital, Ruijin Hospital
- Study start
- 2 Jun 2022 (actual)
- Primary completion
- 31 Mar 2026 (actual)
- Study completion
- 31 Mar 2026 (actual)
- First posted
- 6 Sep 2022 (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 effectiveness and safety of Venetoclax and Decitabin based conditioning regimen followed with post-HSCT Decitabin maintenance therapy in TP53 mutant AML/MDS Patients.
Detailed description
In acute myeloid leukemia and myelodysplastic syndromes, TP53 gene mutation is a poor prognostic factor and a strong indication for hematopoietic stem cell transplantation. However, because of the high relapse rate of myeloid tumors with TP53 mutation, new comprehensive treatment is urgently needed to improve the efficacy of transplantation. Decitabin(DEC) has shown certain efficacy in primary patients with TP53 mutation, and Venetoclax (VEN) and DEC have synergistic effect. Based on this, we hypothesized that DEC and VEN should be added to the conditioning regimen in TP53 mutant AML/MDS patients in order to eliminate malignant clones with P53 mutation as much as possible, and intermittent DEC maintenance therapy should be used to prevent relapse after post-HSCT hematopoietic reconstruction. We intend to conduct a multicenter, single-arm clinical study to evaluate the efficacy and safety of this protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VEN and DEC based conditioning regimen followed with post-HSCT DEC maintenance therapy | Experimental | Drug: VEN and DEC based conditioning regimen, Drug: DEC |
Who may qualify
- Ages
- 12 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- AML or MDS diagnosed according to 2016 WHO criteria with TP53 mutation before enrollment;
- Aged from 12 to 70 years;
- The Eastern Cooperative Oncology Group (ECOG) performance score of 0-2;
- Creatinine clearance rate ≥ 60 mL/min (according to Cockcroft-Gault formula);
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≥ 3× upper limit of normal range (ULN), total bilirubin ≤ 2×ULN;
Key exclusion criteria (6 listed)
- Active autoimmune diseases, such as SLE, rheumatoid arthritis, etc.
- Current active cardiovascular disease with clinically significance, such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease determined by the New York Heart Association (NYHA) functional classification, or a history of myocardial infarction within the 6 months prior to screening;
- Other serious medical conditions (e.g., advanced infection) that may limit the patient's participation in the trial;
- Known human immunodeficiency virus (HIV) infection, or drug-uncontrolled chronic infection of hepatitis B virus (HBV-DNA > 1000IU/ml) or hepatitis C virus (anti-HCV positive);
- Pregnant or lactating women;
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 and DEC based conditioning regimenFor human leukocyte antigen (HLA) matched-donor transplantation, the regimen consisted of venetoclax 400 mg once daily on days -12 to -6, decitabine 20 mg/m² once daily on days -12 to -8, busulfan 0.8 mg/kg every 6 h on days -7 to -4, cyclophosphamide 60 mg/kg once daily on days -3 to -2, and semustine 250 mg/m² on day -1. For haploidentical HCT, the regimen consisted of venetoclax 400 mg once daily on days -14 to -8, decitabine 20 mg/m² once daily on days -14 to -10, cytarabine 2 g/m² once daily on days -9 to -8, busulfan 0.8 mg/kg every 6 h on days -7 to -5, cyclophosphamide 1.8 g/m² once daily on days -4 to -3, and semustine 250 mg/m² on day -2. Patients aged ≥50 years or with an HCT-CI score ≥3 received reduced-toxicity conditioning (RTC), consisting of venetoclax 400 mg once daily on days -12 to -6, decitabine 20 mg/m² once daily on days -12 to -8, fludarabine 30 mg/m² once daily on days -7 to -3, busulfan 0.8 mg/kg every 6 h on days -7 to -5, and semustine 250 mg/m² on day -2. | Drug | VEN and DEC based conditioning regimen followed with post-HSCT DEC maintenance therapy |
| DECIn the time window of 60-120 days after transplantation: DEC 5mg/m2/d for 5 consecutive days every 6 to 8 weeks with a total of 6 courses if there is no severe aGVHD (grade 3 or higher) and the donor chimerism rate of bone marrow blood (STR)>95%. If the MRD turns positive, DLI can be performed. | Drug | VEN and DEC based conditioning regimen followed with post-HSCT DEC maintenance therapy |
Primary outcome
Relapse-free survival (RFS)
Time frame: At Year 1
Relapse-free survival (RFS) was defined as the time from allo-HCT to disease relapse or progression, or death from any cause, whichever occurred first. Relapse or progression was defined as the presence of circulating blasts, >5% blasts in the bone marrow, or extramedullary disease after a prior post-HCT complete response, or as persistent morphologic disease in patients who failed to achieve complete response after HCT.
6 secondary outcomes
Overall survival (OS)
Time frame: At Year 1
Cumulative relapse rate
Time frame: At Year 1
Non-relapse mortality (NRM)
Time frame: At Year 1
Acute graft-versus-host disease (GVHD)
Time frame: At Day 100
Chronic graft-versus-host disease (GVHD)
Time frame: through study completion, an average of 1 year
Adverse effects
Time frame: through study completion, an average of 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites 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 and DEC based conditioning regimen and DEC in people with Hematopoietic Stem Cell Transplantation and TP53, run by Zhejiang University at 4 sites 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 8 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: finished, as of the sponsor's last update on 26 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 4 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relapse-free survival (RFS) (time frame: At Year 1). 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 26 Oct 2026.