An interventional study of Venetoclax and None-placebo in Acute Myeloid Leukemia, High-Risk Acute Myeloid Leukemia and Myelodysplastic Syndromes, sponsored by First Affiliated Hospital of Zhejiang University. Recruiting at 1 site in China. Open to participants aged 12 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-09-02.
Sponsored by First Affiliated Hospital of Zhejiang University · Not applicable, Interventional, and Treatment
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Among 202 studies testing venetoclax in Leukemia, Myeloid, Acute — 116 open to participants now, 144 under way, 21 of them phase 3. venetoclax · Leukemia, Myeloid, Acute.
Among the 202 studies testing venetoclax in Leukemia, Myeloid, Acute, this one is the 45th largest by planned enrollment, is one of 22 that started in 2025, is one of 16 with no phase, reaches primary completion later than 66 of the 143 other live ones, and was the 175th registered.
3,668 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
Browse Leukemia, Myeloid, Acute studies →116 of the 202 studies testing venetoclax in Leukemia, Myeloid, Acute are open now, in 12 countries. Recruiting first, the newest first:
National Cancer Institute (NCI) · Sites in United States, Puerto Rico
Ages 18–59
National Cancer Institute (NCI) · Sites in United States, Puerto Rico
Ages 60+
Virginia Commonwealth University · Sites in United States
Ages 18+
The First Affiliated Hospital of Soochow University · Sites in China
Ages 18+
Beijing 302 Hospital · Sites in China
Ages 18–70
By country, each count listing its studies:
venetoclax: first FDA approval 2016 (VENCLEXTA), first EMA approval 2016. What it is approved for, and how studies give it, is on its page →
Of the 625 Leukemia, Myeloid, Acute studies recruiting now, 363 (58%) set no upper age limit and 222 (36%) stop at 75 or younger; 145 take children under 18; 29 take healthy volunteers.
This study: 12 Years to 60 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
202 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 66 · Phase 2 127 · Phase 3 26 · Phase 4 5
First in 2010; this study’s year is marked. 22 started in 2025.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 138 is above the median of 40 across 3,006 interventional studies indexed under Leukemia, Myeloid, Acute.
Median enrollment 57 across the 202 interventional studies. Of the 46 that completed or were terminated, 15 (33%) have results posted.
This tab as a report to print or save as PDF → With an account.
26 of 202 have an industry lead sponsor. The 10 most frequent:
First Affiliated Hospital of Zhejiang University is the lead sponsor of 264 studies on the registry; 145 are open to participants now.
16 studies in Leukemia, Myeloid, Acute by First Affiliated Hospital of Zhejiang University: 15 open now, 15 under way, 2 of them phase 3. Of 1 finished, 0 (0%) have results posted.
Among the 16 studies in Leukemia, Myeloid, Acute by First Affiliated Hospital of Zhejiang University, this one is the 4th largest by planned enrollment, is one of 5 that started in 2025, is one of 2 with no phase, reaches primary completion later than 7 of the 14 other live ones, and was the 10th registered.
First Affiliated Hospital of Zhejiang University’s Leukemia, Myeloid, Acute studies by the year they started, since 2022.
38 of the studies testing venetoclax in Leukemia, Myeloid, Acute that are under way reach primary completion within a year, 5 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07264010Phase 3Nanfang Hospital, Southern Medical UniversitySorafenib Combined With Venetoclax as Pre-emptive Therapy Strategy for MRD+ AML: a Prospective, Single-arm, Multicenter Clinical Study | 2026-12-30 |
| NCT07486479Phase 3The First Affiliated Hospital of Soochow UniversityVenetoclax, Azacitidine, and Mitoxantrone Hydrochloride Liposome Versus Idarubicin and Cytarabine in Newly Diagnosed AML | 2026-12-31 |
| NCT05183035Phase 3PedAL BCU, LLCVenetoclax in Children With Relapsed Acute Myeloid Leukemia (AML) | 2027-02 |
| NCT03844048Phase 3AbbVieAn Extension Study of Venetoclax for Subjects Who Have Completed a Prior Venetoclax Clinical Trial | 2027-08 |
| NCT07514936Phase 3Chinese PLA General HospitalVenetoclax-Azacitidine in Combination With Chidamide and CAG in Fit Older Patients With Acute Myeloid Leukaemia | 2027-09-30 |
Add all 38 to your calendar (.ics) → With an account.
Among the 202 studies testing venetoclax in Leukemia, Myeloid, Acute, from their registry updates:
Among First Affiliated Hospital of Zhejiang University’s 16 in Leukemia, Myeloid, Acute:
This tab as a report to print or save as PDF → With an account.
This is a prospective, multicenter, randomized controlled trial designed to evaluate the efficacy and safety of venetoclax-enhanced BUCY (Ven-BUCY) conditioning compared to the standard BUCY regimen in patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible participants aged 12 to 60 years will be randomized 1:1 to receive either Ven-BUCY or standard BUCY conditioning. The primary endpoint is relapse-free survival (RFS) at two years post-transplant. Secondary outcomes include overall survival, relapse rate, non-relapse mortality, measurable residual disease (MRD), and treatment-related adverse events. The study aims to improve post-transplant outcomes by deepening disease remission through the addition of venetoclax, a BCL-2 inhibitor known to target leukemia stem cells and enhance chemotherapy sensitivity.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative therapy for high-risk acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). However, post-transplant relapse remains the leading cause of treatment failure, especially among patients receiving matched sibling or unrelated donor transplants. While myeloablative conditioning (MAC) regimens like BUCY (busulfan + cyclophosphamide) offer stronger anti-leukemic effects compared to reduced-intensity regimens, the relapse rate in high-risk myeloid neoplasms remains unacceptably high, partly due to residual leukemia stem cells (LSCs).
Venetoclax, a selective BCL-2 inhibitor, has shown synergistic effects when combined with hypomethylating agents or intensive chemotherapy. It improves remission depth and targets chemotherapy-resistant LSCs. Emerging data suggest that venetoclax may also enhance graft-versus-leukemia (GVL) effects without significantly increasing the risk of graft-versus-host disease (GVHD).
This investigator-initiated, open-label, two-arm, randomized controlled trial will enroll 138 patients aged 12-60 years with high-risk AML or MDS across six transplant centers in China. Patients will be stratified by disease (AML vs. MDS) and randomized (1:1) to receive either:
Standard BUCY regimen: Busulfan (0.8 mg/kg q6h on day -7 to -4), Cyclophosphamide (60 mg/kg/day on day -3 and -2), and MeCCNU (250 mg/m² on day -1), with optional ATG for donors/recipients >40 years.
Ven-BUCY regimen: Venetoclax (400 mg/day or 360 mg/m²/day from day -14 to -8) in addition to the standard BUCY components, with similar ATG guidance. Antifungal prophylaxis and venetoclax dose adjustment (e.g., to 100 mg with posaconazole) will follow local guidelines.
Patients will be followed weekly during the first month post-transplant, monthly for 6 months, and every 3 months thereafter until 3 years post-enrollment. The primary endpoint is 2-year relapse-free survival (RFS). Secondary endpoints include overall survival (OS), non-relapse mortality (NRM), relapse rate, MRD clearance, and adverse events graded by CTCAE v5.0.
This study seeks to determine whether adding venetoclax to a standard myeloablative regimen can enhance anti-leukemic efficacy and improve long-term outcomes without increasing transplant-related toxicity.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
High-risk MDS as defined by at least one of the following:
High-risk AML as defined by at least one of the following:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants in this arm will receive a venetoclax-enhanced BUCY conditioning regimen before undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). 1. Venetoclax is administered from day -14 to -8 (400 mg/day for patients ≥14 years; 360 mg/m²/day for patients aged 12-14 years). 2. Busulfan (0.8 mg/kg every 6 hours, days -7 to -4). 3. Cyclophosphamide (60 mg/kg/day, days -3 and -2). 4. MeCCNU (250 mg/m² on day -1). Antithymocyte globulin (ATG) may be added for donor or recipient age \>40 years. Venetoclax dose is reduced if co-administered with strong CYP3A4 inhibitors such as posaconazole.
Drug: Venetoclax · Drug: Busulfan (BU)
Participants in this arm will receive the standard BUCY myeloablative conditioning regimen prior to allo-HSCT. 1. Busulfan (0.8 mg/kg every 6 hours, days -7 to -4). 2. Cyclophosphamide (60 mg/kg/day, days -3 and -2). 3. MeCCNU (250 mg/m² on day -1). ATG may be included in cases where the donor or recipient is over 40 years old. No venetoclax is used in this arm.
Other: None-placebo · Drug: Busulfan (BU)
Venetoclax is administered orally at 400 mg/day for participants ≥14 years or 360 mg/m²/day for those aged 12-14 years, from day -14 to -8 before allogeneic hematopoietic stem cell transplantation (allo-HSCT). Dose is adjusted to 100 mg/day (or 90 mg/m²/day for pediatric patients) if used with strong CYP3A4 inhibitors such as posaconazole.
Also known as: ABT-199, Venclexta
Participants in this arm will not receive venetoclax as part of their conditioning regimen. They will undergo standard myeloablative conditioning with BUCY (busulfan, cyclophosphamide, and MeCCNU), prior to allogeneic hematopoietic stem cell transplantation. This arm serves as the active comparator to evaluate the addition of venetoclax in the experimental arm.
BUCY group (Control group): Busulfan (BU 0.8 mg/kg/q6h, days -7 to -4), Cyclophosphamide (CTX 60 mg/kg/day, days -3 to -2), and MeCCNU (250 mg/m²/day, day -1). For recipients or donors over 40 years of age, ATG (Thymoglobulin, total dose 2.5-4.5 mg/kg, divided over days -3 to -1) may be added.
Also known as: Cyclophosphamide, MeCCNU, Busulfan
Relapse-Free Survival (RFS)
Relapse-Free Survival (RFS) is defined as the time from the date of allogeneic hematopoietic stem cell transplantation (allo-HSCT) to the first documented relapse of the primary disease or death from any cause, whichever occurs first. Participants who remain alive and relapse-free will be censored at the time of last follow-up.
Time frame: From the date of transplantation until relapse or death from any cause, assessed up to 24 months
Overall Survival (OS)
Overall survival (OS) is defined as the time from the date of transplantation to death from any cause. Patients who are alive at the last follow-up will be censored. Survival curves will be estimated using the Kaplan-Meier method.
Time frame: Up to 36 months post-transplantation
Non-Relapse Mortality (NRM)
Non-relapse mortality (NRM) is defined as death from any cause other than disease relapse after transplantation. Competing risk analysis will be used to assess NRM incidence between groups.
Time frame: Up to 36 months post-transplantation
Relapse Rate
Relapse rate refers to the cumulative incidence of disease recurrence post-transplantation, assessed through bone marrow evaluation, flow cytometry, and molecular markers. Competing risk models will be applied for statistical analysis.
Time frame: Up to 36 months post-transplantation
Measurable Residual Disease (MRD) Status
MRD will be assessed by multiparameter flow cytometry and/or molecular techniques at scheduled timepoints after transplantation to evaluate minimal residual leukemia. MRD positivity and conversion dynamics will be compared between groups.
Time frame: Assessed monthly during the first 6 months, and then every 3 months until 24 months post-transplantation
Treatment-Related Adverse Events
Incidence and severity of treatment-related adverse events, including hematologic and non-hematologic toxicities, will be evaluated and graded according to NCI-CTCAE v5.0. Events will be compared between the VEN-BUCY and BUCY groups.
Time frame: From start of conditioning until 28 days after transplantation, and during follow-up up to 3 months
Incidence of Acute Graft-versus-Host Disease (aGVHD)
Acute GVHD will be assessed and graded based on MAGIC (Mount Sinai Acute GVHD International Consortium) criteria. The cumulative incidence of grade II-IV and grade III-IV aGVHD will be compared between the VEN-BUCY and BUCY groups.
Time frame: From day of stem cell infusion to day 100 post-transplantation
Incidence of Chronic Graft-versus-Host Disease (cGVHD)
Chronic GVHD will be evaluated according to the 2014 NIH consensus criteria. The cumulative incidence of overall and moderate-to-severe cGVHD will be recorded and compared between the two groups.
Time frame: From day 100 post-transplantation to 36 months
Plan to share: Yes
Supporting information: Study protocol
No publications or documents are linked to this record.
Started Aug 2025 (actual). Primary completion is estimated for Aug 2027, 24 months after the start.
Of 132 non-industry interventional studies with no phase under Leukemia, Myeloid, Acute, half reached primary completion within 49 months of starting (the median), counting those still running; a quarter within 26 and three quarters within 128. This study's planned 24 months is below that.
Counting every sponsor, half of 139 studies reached it within 47 months; a quarter within 25 and three quarters within 123.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Studies of the drug tested here: Venetoclax
First Affiliated Hospital of Zhejiang University →