Venetoclax-Enhanced BUCY vs. Standard BUCY Conditioning in High-Risk AML and MDS Patients Undergoing Allo-HSCT (Ven-BUCY Study)

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.

NCT Number	NCT07183878
Lead Sponsor	First Affiliated Hospital of Zhejiang University Collaborators: The First Affiliated Hospital of Zhengzhou University, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Tongji Hospital, The Children's Hospital of Zhejiang University School of Medicine, Ruijin Hospital
Conditions	Acute Myeloid Leukemia, High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndromes, High-Risk Myelodysplastic Syndromes
Enrollment	138 participants
Start Date	2025-08-20
Primary Completion	2027-08-20 (estimated)
Study Completion	2028-08-20 (estimated)
Updated on ClinicalTrials.gov	2025-09-19