Hypoplastic Acute Myeloid LeukemiaNCT07635485
VA Induction and Bridging Transplantation for Hypoplastic/Secondary AML
A study testing Venetoclax (VEN), Azacitidine (AZA) and Allo-HSCT in people with Hypoplastic Acute Myeloid Leukemia and Secondary AML (Adult), run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
The purpose of this study is to explore the efficacy and safety of venetoclax combined with azacitidine（VA) induction followed by bridging allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of hypoplastic and secondary acute myeloid leukemia (AML). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypoplastic Acute Myeloid Leukemia, Secondary AML (Adult)
- What is being tested?
- Venetoclax (VEN), Azacitidine (AZA) and Allo-HSCT
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2026to 31 Aug 2029
- Phase
- Interventional
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years.
- Previously untreated acute myeloid leukemia (AML) diagnosed according to the 2022 ELN guidelines. Patients with isolated extramedullary disease (i.e., no evidence of AML in bone marrow or peripheral blood) are not eligible.
- Bone marrow biopsy demonstrating cellularity <20% or concurrent myelofibrosis; Or a prior history of an antecedent hematologic disorder, radiotherapy/chemotherapy-related history, or presence of myelodysplasia-related changes (AML-MRC according to WHO-HEAM5).
- The patient is deemed suitable for allogeneic hematopoietic stem cell transplantation as assessed by the treating physician.
- Adequate organ function, defined as follows: a. Good liver function: serum total bilirubin ≤2.0 × upper limit of normal (ULN); if considered due to Gilbert's disease or leukemia, serum total bilirubin <3.0 × ULN. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemia. b. Good renal function: serum creatinine ≤2.0 × ULN or creatinine clearance >30 mL/min calculated using the Cockcroft-Gault formula. c. No history of chronic lung disease and no dyspnea. Otherwise, documented diffusing capacity of the lung for carbon monoxide ≤40% (adjusted for hemoglobin if available) and forced expiratory volume in 1 second/forced vital capacity ≥50%.
- ECOG performance status score 0-2.
- Ability to understand and voluntarily sign informed consent.
- Women of childbearing potential must have a negative serum pregnancy test before initiation of study treatment.
Who cannot join
- Prior treatment for AML, except non-cytotoxic therapy given to stabilize disease.
- White blood cell count ≥10×10⁹/L, or presence of proliferation-associated gene mutations such as FLT3.
- Favorable risk group according to the 2022 ELN prognostic stratification, e.g., t(8;21), inv(16)/t(16;16), NPM1 mutation, or CEBPA bZIP in-frame mutation.
- No suitable stem cell donor available.
- Acute promyelocytic leukemia (APL).
- Clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia.
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- Life-threatening immediate complications of leukemia, such as uncontrolled bleeding, hypoxic pneumonia, sepsis, and/or disseminated intravascular coagulation (DIC). Expected survival <12 weeks.
- Prior allogeneic hematopoietic stem cell transplantation for a hematologic disorder.
- Current use of strong CYP3A4 inducers or narrow-therapeutic-window CYP3A4 substrates; enrollment is allowed only if these drugs can be switched to alternatives ≥5 half-lives before the first dose of study treatment.
- Active, uncontrolled systemic fungal, bacterial, or viral infection despite appropriate antibiotic, antiviral, or other therapy.
- Known infection with human immunodeficiency virus (HIV) or active hepatitis B virus (HBV) or hepatitis C virus (HCV) that cannot be controlled by therapy.
- Another active malignancy, unless the patient has been disease-free for ≥5 years before initiation of study treatment. However, patients with the following history/concurrent conditions or similar indolent cancers are eligible: Basal cell or squamous cell carcinoma of the skin Carcinoma in situ of the cervix Carcinoma in situ of the breast Prostate cancer found incidentally on histology.
- Significant active cardiac disease within 6 months before initiation of study treatment, including New York Heart Association (NYHA) Class III or IV congestive heart failure, myocardial infarction, unstable angina, and/or stroke.
- Uncontrolled hypertension (systolic blood pressure >180 mmHg or diastolic blood pressure >100 mmHg).
- Dysphagia, short-bowel syndrome, gastroparesis, or other conditions that limit oral intake or gastrointestinal absorption.
- Known history of progressive multifocal leukoencephalopathy (PML).
- Known hypersensitivity to any component of venetoclax or azacitidine.
- Female patient who is pregnant or breastfeeding.
- Any other medical or psychological condition that, in the investigator's opinion, could interfere with the patient's ability to sign informed consent or participate in the 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
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215000
About this study
Official title: A Multicenter, Single-arm Clinical Study of Venetoclax Combined With Azacitidine Induction and Bridging to Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Hypoplastic or Secondary Acute Myeloid Leukemia
The purpose of this study is to explore the efficacy and safety of venetoclax combined with azacitidine（VA) induction followed by bridging allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of hypoplastic and secondary acute myeloid leukemia (AML).
Read the detailed description
Under the traditional intensive chemotherapy model, patients with hypoplastic or secondary acute myeloid leukemia (AML) often have poor bone marrow reserve and prolonged post-chemotherapy cytopenia, leading to high early mortality, low remission rates, and short median survival. To address this, the use of venetoclax combined with a hypomethylating agent (the VA regimen) as induction therapy significantly improves complete remission (CR) and overall survival (OS). Subsequent bridging to allogeneic hematopoietic stem cell transplantation (allo-HSCT) as consolidation further enhances OS and relapse-free survival (RFS). This combined strategy demonstrates a clear synergistic effect: the VA regimen effectively increases the transplantation rate, and for patients with relapsed/refractory (R/R) AML, a low-intensity regimen followed by bridging transplantation is non-inferior to traditional intensive chemotherapy, achieving a "1+1>2" therapeutic benefit. Therefore, the investigators conducted a prospective study to evaluate the efficacy and safety of VA regimen induction followed by allo-HSCT in patients with hypoplastic and secondary AML.
- Being tested
- Venetoclax (VEN), Azacitidine (AZA), Allo-HSCT
- Keywords
- Venetoclax, Azacitidine, Allo-HSCT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Venetoclax (VEN)Drug
Venetoclax was administered at 100 mg on day 1, 200 mg on day 2, and 400 mg on days 3-28 of cycle 1.
Azacitidine (AZA)Drug
Azacitidine was administered at 75 mg/m² on days 1-7 of cycle 1.
Allo-HSCTProcedure or surgery
All patients proceeded directly to allogeneic HSCT after cycle 1, regardless of remission. Myeloablative or intensified conditioning was preferred; reduced-intensity conditioning was allowed for intolerant patients. Donor source and transplant type were not restricted.
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | VA induction+Allo-HSCT consolidation Receives: Drug: Venetoclax (VEN); Drug: Azacitidine (AZA); Procedure: Allo-HSCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival (OS)
Time from the date of diagnosis to death due to any cause.
Measured: 2 year
Secondary outcomes (6)
Complete Remission Rate（CRR）
Bone marrow blasts <5%, absence of extramedullary disease, no circulating blasts, peripheral blood neutrophil count ≥1.0×10⁹/L, platelet count ≥100×10⁹/L, independent of transfusion or growth factor support.
Measured: Day 28 of induction therapy and 56 days post-transplantation
Composite Complete Remission Rate (CCRR)
The proportion of patients achieving complete remission (CR), complete remission with incomplete hematologic recovery (CRi), or complete remission with partial hematologic recovery (CRh).
Measured: Day 28 of induction therapy and 56 days post-transplantation
Disease-free Survival (DFS)
Time from achieving disease remission to disease relapse or death from any cause.
Measured: 2 year
GVHD-free and Relapse-free Survival (GRFS)
Time from transplant date to the first occurrence of grade III-IV acute GVHD, chronic GVHD requiring systemic treatment, disease relapse, or death from any cause.
Measured: 2 year
Cumulative Incidence of Relapse (CIR)
Time from transplant date to disease relapse.
Measured: 2 year
Non-relapse Mortality (NRM)
Time from transplant date to death due to non-relapse/progression causes.
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 posted9 Jun 2026
- Study start1 Jun 2026 (estimated)
- Primary completion31 May 2029 (estimated)
- Study completion31 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypoplastic Acute Myeloid LeukemiaStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Nanfang Hospital, Southern Medical University, Ruijin Hospital
- Responsible party
- Sponsor
- Investigators
- Suning Chen, Doctor, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07635485
- Sponsor's protocol ID
- 2026531
- Other IDs
- 09021420250003
- Status last verified
- May 2026
- First submitted
- 1 Jun 2026
- Last updated
- 9 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 9 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Single-arm Clinical Study of Venetoclax Combined With Azacitidine Induction and Bridging to Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Hypoplastic or Secondary Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07635485
- Acronym / sponsor ID
- 2026531
- Keywords
- Venetoclax, Azacitidine, Allo-HSCT
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Collaborators
- Nanfang Hospital, Southern Medical University, Ruijin Hospital
- Study start
- 1 Jun 2026 (estimated)
- Primary completion
- 31 May 2029 (estimated)
- Study completion
- 31 Aug 2029 (estimated)
- First posted
- 9 Jun 2026 (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 explore the efficacy and safety of venetoclax combined with azacitidine（VA) induction followed by bridging allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of hypoplastic and secondary acute myeloid leukemia (AML).
Detailed description
Under the traditional intensive chemotherapy model, patients with hypoplastic or secondary acute myeloid leukemia (AML) often have poor bone marrow reserve and prolonged post-chemotherapy cytopenia, leading to high early mortality, low remission rates, and short median survival. To address this, the use of venetoclax combined with a hypomethylating agent (the VA regimen) as induction therapy significantly improves complete remission (CR) and overall survival (OS). Subsequent bridging to allogeneic hematopoietic stem cell transplantation (allo-HSCT) as consolidation further enhances OS and relapse-free survival (RFS). This combined strategy demonstrates a clear synergistic effect: the VA regimen effectively increases the transplantation rate, and for patients with relapsed/refractory (R/R) AML, a low-intensity regimen followed by bridging transplantation is non-inferior to traditional intensive chemotherapy, achieving a "1+1>2" therapeutic benefit. Therefore, the investigators conducted a prospective study to evaluate the efficacy and safety of VA regimen induction followed by allo-HSCT in patients with hypoplastic and secondary AML.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: Venetoclax (VEN), Drug: Azacitidine (AZA), Procedure: Allo-HSCTVA induction+Allo-HSCT consolidation |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age ≥18 years.
- Previously untreated acute myeloid leukemia (AML) diagnosed according to the 2022 ELN guidelines. Patients with isolated extramedullary disease (i.e., no evidence of AML in bone marrow or peripheral blood) are not eligible.
- Bone marrow biopsy demonstrating cellularity <20% or concurrent myelofibrosis; Or a prior history of an antecedent hematologic disorder, radiotherapy/chemotherapy-related history, or presence of myelodysplasia-related changes (AML-MRC according to WHO-HEAM5).
- The patient is deemed suitable for allogeneic hematopoietic stem cell transplantation as assessed by the treating physician.
- Adequate organ function, defined as follows: a. Good liver function: serum total bilirubin ≤2.0 × upper limit of normal (ULN); if considered due to Gilbert's disease or leukemia, serum total bilirubin <3.0 × ULN. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemia. b. Good renal function: serum creatinine ≤2.0 × ULN or creatinine clearance >30 mL/min calculated using the Cockcroft-Gault formula. c. No history of chronic lung disease and no dyspnea. Otherwise, documented diffusing capacity of the lung for carbon monoxide ≤40% (adjusted for hemoglobin if available) and forced expiratory volume in 1 second/forced vital capacity ≥50%.
Key exclusion criteria (19 listed)
- Prior treatment for AML, except non-cytotoxic therapy given to stabilize disease.
- White blood cell count ≥10×10⁹/L, or presence of proliferation-associated gene mutations such as FLT3.
- Favorable risk group according to the 2022 ELN prognostic stratification, e.g., t(8;21), inv(16)/t(16;16), NPM1 mutation, or CEBPA bZIP in-frame mutation.
- No suitable stem cell donor available.
- Acute promyelocytic leukemia (APL).
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 was administered at 100 mg on day 1, 200 mg on day 2, and 400 mg on days 3-28 of cycle 1. | Drug | A |
| Azacitidine (AZA)Azacitidine was administered at 75 mg/m² on days 1-7 of cycle 1. | Drug | A |
| Allo-HSCTAll patients proceeded directly to allogeneic HSCT after cycle 1, regardless of remission. Myeloablative or intensified conditioning was preferred; reduced-intensity conditioning was allowed for intolerant patients. Donor source and transplant type were not restricted. | Procedure or surgery | A |
Primary outcome
Overall Survival (OS)
Time frame: 2 year
Time from the date of diagnosis to death due to any cause.
6 secondary outcomes
Complete Remission Rate（CRR）
Time frame: Day 28 of induction therapy and 56 days post-transplantation
Composite Complete Remission Rate (CCRR)
Time frame: Day 28 of induction therapy and 56 days post-transplantation
Disease-free Survival (DFS)
Time frame: 2 year
GVHD-free and Relapse-free Survival (GRFS)
Time frame: 2 year
Cumulative Incidence of Relapse (CIR)
Time frame: 2 year
Non-relapse Mortality (NRM)
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 9 Jun 2026.
- Individual participant dataSharing planned: No
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.
Suning Chen, Doctor principal investigator
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 study testing Venetoclax (VEN), Azacitidine (AZA) and Allo-HSCT in people with Hypoplastic Acute Myeloid Leukemia and Secondary AML (Adult), 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 8 inclusion and 19 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 9 Jun 2026. 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.
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 Zhen Shen, Doctor (0512-67976802, zhenshen96@sina.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (OS) (time frame: 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 9 Jun 2026; AzTrials last refreshed its copy on 6 Nov 2026.