Adding venetoclax before stem cell transplant for high-risk leukemia and MDS
Tests adding venetoclax to chemotherapy before stem cell transplant in people with high-risk leukemia.
Plain-language title generated from the registry record
Registry title: Venetoclax-Enhanced BUCY vs. Standard BUCY Conditioning in High-Risk AML and MDS Patients Undergoing Allo-HSCT (Ven-BUCY Study)
Key facts
- Where
- Hangzhou, ChinaSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 60 years old?
Source: NCT07183878, who can take part
Where in China
- The First Affiliated Hospital of Zhejiang University School of MedicineHangzhou, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- First Affiliated Hospital of Zhejiang University
- Trial code
- NCT07183878
- Record updated
- 2 September 2026
- Original description
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.Read full original descriptionShow less
A Prospective, Multicenter, Randomized Controlled Study Comparing Venetoclax-Enhanced BUCY With Standard BUCY Conditioning in High-Risk AML and MDS Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
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.
Inclusion: Diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) according to 2022 WHO classification; Age between 12 and 60 years; High-risk MDS as defined by at least one of the following:; IPSS intermediate-2/high risk or IPSS-R intermediate/high/very high risk; TP53 mutation; RAS pathway mutation (e.g., NRAS, KRAS, PTPN11, CBL, NF1, RIT1, FLT3, KIT); Therapy-related MDS; High-risk AML as defined by at least one of the following:; TP53, RUNX1, or ASXL1 mutation; t(6;9)(p23;q34.1)/DEK-NUP214; KMT2A rearrangement; BCR-ABL1 fusion; inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2); -5/del(5q), -7, -17/abn(17p); Complex or monosomal karyotype; FLT3-ITD high with wild-type NPM1; Initial WBC ≥ 10×10\^9/L; Secondary AML with history of MDS/MPN or therapy-related AML; AML with specific mutations (SRSF2, SF3B1, U2AF1, ZRSR2, ASXL1, EZH2, BCOR, STAG2); MRD positive before transplantation; For AML: must have achieved CR or CRi prior to transplantation; for MDS: bone marrow blasts < 20%; Availability of a matched related or unrelated donor (10/10 or 9/10 HLA match); ECOG performance status 0-2; Creatinine clearance ≥ 60 mL/min (Cockcroft-Gault); AST/ALT ≤ 3 × ULN and total bilirubin ≤ 2 × ULN; LVEF ≥ 50% by echocardiogram; Life expectancy > 8 weeks; Willingness to use effective contraception methods during and for a specified period after the study; Signed informed consent.
Exclusion: More than one prior hematopoietic stem cell transplantation; History of grade ≥ 3 non-hematologic adverse reaction to prior venetoclax therapy; Receipt of chemotherapy (except hydroxyurea/dexamethasone) or radiotherapy within 14 days before study treatment; Ongoing use of BCR-ABL1, IDH, or FLT3 inhibitors without proper washout (≥ 7 days); Uncontrolled cardiovascular disease or New York Heart Association class III/IV heart failure; Other severe comorbid conditions that may interfere with study participation; Known HIV infection or uncontrolled active hepatitis B or C; Pregnant or breastfeeding women; Inability to understand the study protocol or provide informed consent.