Relapse LeukemiaPhase 2NCT07091006
Efficacy and Safety of VAH as a Bridging Regimen to Allo-HCT in Relapsed/Refractory AML
A Phase 2 study testing VAH in people with Relapse Leukemia, run by Ruijin Hospital at 1 site in China.
Trial summary
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only treatment that offers a possible cure for relapsed/refractory AML. Currently, the optimal preallo-HCT bridging regimen for relapsed/refractory AML patients is unclear. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Relapse Leukemia
- What is being tested?
- VAH
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2025to 1 May 2028
- 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 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged 14 to 70 years (inclusive), regardless of gender;
- The diagnosis of AML was confirmed on the basis of bone marrow cytomorphology, immunophenotyping and chromosomal and molecular biology tests;
- Relapsed AML: After achieving complete remission (CR), the reappearance of leukemic cells in peripheral blood, or ≥5% blasts in bone marrow (excluding other causes such as bone marrow regeneration after consolidation chemotherapy), or extramedullary infiltration by leukemic cells.
- Refractory AML: Newly diagnosed cases that are unresponsive after two courses of standard induction therapy; patients who relapse within 12 months after consolidation/intensification therapy post-CR; patients who relapse after 12 months but fail to respond to conventional chemotherapy; patients with two or more relapses; or those with persistent extramedullary leukemia.
- With RUNX1::RUNX1T1 AML: Positive measurable residual disease (MRD) on bone marrow flow evaluation after the second consolidation therapy and/or less than a 3-log decrease in the RUNX1::RUNX1T1 fusion gene and diagnostic baseline values;
- ECOG score ≤2; HCT-CI score <3; Aspartate aminotransferase (AST) ≤ 3 times ULN (upper limit of normal, ULN); Alanine aminotransferase (ALT) ≤ 3x ULN; Total serum bilirubin ≤ 1.5 times the upper limit of normal ULN unless the patient has documented Gilbert syndrome; patients with Gilbert-Meulengracht syndrome with bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included; Serum creatinine ≤ 1.5 times ULN or creatinine clearance ≥ 60 mL/min; Coagulation function: International Normalised Ratio (INR) ≤ 1.5 x ULN, Activated Partial Thromboplastin Time (APTT) ≤ 1.5 x ULN;
- Left ventricular ejection fraction (LVEF) ≥50%;
Who cannot join
- Allergies or contraindications to any of the drugs in the protocol;
- Currently have clinically significant active cardiovascular disease such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- Serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes);
- Active autoimmune diseases such as SLE, rheumatoid arthritis, etc;
- Patients with neurological or psychiatric disorders;
- The patient is pregnant or breastfeeding;
- Those who are unable to understand or comply with the study protocol or are unable to sign the informed consent form.
- Other conditions that, in the opinion of the investigator, make the patient otherwise unsuitable for participation in this study;
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
Ruijin Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality
About this study
Official title: A Prospective Study on the Efficacy and Safety of Venetoclax, Azacitidine, and Homoharringtonine (VAH) Combination as a Bridging Regimen to Allogeneic Hematopoietic Stem Cell Transplantation in Relapsed/Refractory Acute Myeloid Leukemia
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only treatment that offers a possible cure for relapsed/refractory AML. Currently, the optimal preallo-HCT bridging regimen for relapsed/refractory AML patients is unclear. Venetoclax-based regimens, including Venetoclax + demethylating agents (HMA) , Venetoclax + HMA + other drugs and Venetoclax-based multidrug combinations as a bridging regimen improves response rate and post-transplant survival in relapsed/refractory AML patients. Therefore, the investigators conduct a prospective single-centre clinical study to evaluate the efficacy and safety of VAH as a transplant bridging regimen for relapsed/refractory AML.
Read the detailed description
Relapsed/refractory acute myeloid leukaemia has an extremely poor prognosis, and allogeneic haematopoietic stem cell transplantation is the only treatment that offers the possibility of a cure for relapsed/refractory AML. Currently, the optimal pre-allo-HCT bridging regimen for relapsed/refractory AML patients is unclear. Previous studies have found that venetoclax and Azacitidine combined with homoharringtonine (VAH) bridging allogeneic transplantation for relapsed/refractory AML has a post-transplantation CRc of 78% and a 1-year OS of 85%, which is superior to other bridging regimens, and we have also found that VAH has a better safety and efficacy as a pre-transplantation bridging in our previous study. Therefore, the investigators propose to conduct a prospective single-centre clinical study to evaluate the efficacy and safety of VAH as a bridging regimen for transplantation in relapsed/refractory AML.
The aim of the study is assessment of the efficacy and safety of allogeneic haematopoietic stem cell transplantation for relapsed/refractory acute myeloid leukaemia in combination with venetoclax and Azacitidine in combination with homoharringtonine.
- Conditions
- Relapse Leukemia
- Being tested
- VAH
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VAHDrug
For R/R AML patients, VAH bridging to conditioning regimen for allo-HCT.
| Study group | Role | What they receive |
|---|---|---|
| Experimental arm | Experimental | Patients with relapsed/refractory acute myeloid leukaemia eligible for enrolment should be bridged with venetoclax, azacitidine, in combination with homoharringtonine before allo-HCT. Receives: Drug: VAH |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-year post-transplant relapse rate
relapse
Measured: through study completion, an average of 2 year
Secondary outcomes (2)
2-year event-free survival after transplantation
MRD positivity, relapse, death
Measured: through study completion, an average of 2 year
Overall survival at 2 years after transplantation
survival
Measured: through study completion, an average of 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2025
- Study start1 Aug 2025 (estimated)
- Primary completion1 May 2028 (estimated)
- Study completion1 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapse LeukemiaStudies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe 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
- 44 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Xiaoxia HU, Principal investigator, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07091006
- Sponsor's protocol ID
- RJ-BMT-013
- Status last verified
- Jul 2025
- First submitted
- 7 Jul 2025
- Last updated
- 29 Jul 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study on the Efficacy and Safety of Venetoclax, Azacitidine, and Homoharringtonine (VAH) Combination as a Bridging Regimen to Allogeneic Hematopoietic Stem Cell Transplantation in Relapsed/Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07091006
- Acronym / sponsor ID
- RJ-BMT-013
- Conditions
- Relapse Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 1 Aug 2025 (estimated)
- Primary completion
- 1 May 2028 (estimated)
- Study completion
- 1 May 2028 (estimated)
- First posted
- 29 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.
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only treatment that offers a possible cure for relapsed/refractory AML. Currently, the optimal preallo-HCT bridging regimen for relapsed/refractory AML patients is unclear. Venetoclax-based regimens, including Venetoclax + demethylating agents (HMA) , Venetoclax + HMA + other drugs and Venetoclax-based multidrug combinations as a bridging regimen improves response rate and post-transplant survival in relapsed/refractory AML patients. Therefore, the investigators conduct a prospective single-centre clinical study to evaluate the efficacy and safety of VAH as a transplant bridging regimen for relapsed/refractory AML.
Detailed description
Relapsed/refractory acute myeloid leukaemia has an extremely poor prognosis, and allogeneic haematopoietic stem cell transplantation is the only treatment that offers the possibility of a cure for relapsed/refractory AML. Currently, the optimal pre-allo-HCT bridging regimen for relapsed/refractory AML patients is unclear. Previous studies have found that venetoclax and Azacitidine combined with homoharringtonine (VAH) bridging allogeneic transplantation for relapsed/refractory AML has a post-transplantation CRc of 78% and a 1-year OS of 85%, which is superior to other bridging regimens, and we have also found that VAH has a better safety and efficacy as a pre-transplantation bridging in our previous study. Therefore, the investigators propose to conduct a prospective single-centre clinical study to evaluate the efficacy and safety of VAH as a bridging regimen for transplantation in relapsed/refractory AML.
The aim of the study is assessment of the efficacy and safety of allogeneic haematopoietic stem cell transplantation for relapsed/refractory acute myeloid leukaemia in combination with venetoclax and Azacitidine in combination with homoharringtonine.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental arm | Experimental | Drug: VAHPatients with relapsed/refractory acute myeloid leukaemia eligible for enrolment should be bridged with venetoclax, azacitidine, in combination with homoharringtonine before allo-HCT. |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Aged 14 to 70 years (inclusive), regardless of gender;
- The diagnosis of AML was confirmed on the basis of bone marrow cytomorphology, immunophenotyping and chromosomal and molecular biology tests;
- Relapsed AML: After achieving complete remission (CR), the reappearance of leukemic cells in peripheral blood, or ≥5% blasts in bone marrow (excluding other causes such as bone marrow regeneration after consolidation chemotherapy), or extramedullary infiltration by leukemic cells.
- Refractory AML: Newly diagnosed cases that are unresponsive after two courses of standard induction therapy; patients who relapse within 12 months after consolidation/intensification therapy post-CR; patients who relapse after 12 months but fail to respond to conventional chemotherapy; patients with two or more relapses; or those with persistent extramedullary leukemia.
- With RUNX1::RUNX1T1 AML: Positive measurable residual disease (MRD) on bone marrow flow evaluation after the second consolidation therapy and/or less than a 3-log decrease in the RUNX1::RUNX1T1 fusion gene and diagnostic baseline values;
Key exclusion criteria (8 listed)
- Allergies or contraindications to any of the drugs in the protocol;
- Currently have clinically significant active cardiovascular disease such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- Serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes);
- Active autoimmune diseases such as SLE, rheumatoid arthritis, etc;
- Patients with neurological or psychiatric disorders;
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 |
|---|---|---|
| VAHFor R/R AML patients, VAH bridging to conditioning regimen for allo-HCT. | Drug | Experimental arm |
Primary outcome
2-year post-transplant relapse rate
Time frame: through study completion, an average of 2 year
relapse
2 secondary outcomes
2-year event-free survival after transplantation
Time frame: through study completion, an average of 2 year
Overall survival at 2 years after transplantation
Time frame: through study completion, an average of 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 29 Jul 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.
Xiaoxia HU principal investigator
Ruijin Hospital
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 VAH in people with Relapse Leukemia, run by Ruijin Hospital 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 7 inclusion and 8 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 29 Jul 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 Xiaoxia HU (02164370045, hu_xiaoxia@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-year post-transplant relapse rate (time frame: through study completion, an average of 2 year). 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 29 Jul 2025.