Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN

Summary

This clinical trial involves individuals who have been diagnosed with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Myelomonocytic Leukemia (CMML), or MDS/myeloproliferative neoplasm-unclassifiable (MDS/MPN-unclassifiable) and are planning to have an allogeneic hematopoietic stem cell transplant ("bone marrow transplant"). The goal of this research study is to (1) test the safety of adding the study drug, Venetoclax, to a standard of care conditioning regimen for bone marrow transplantation as a possible means of eliminating residual (left-over) disease prior to transplant, (2) to test the safety of combination Venetoclax and azacitidine as "maintenance therapy" after transplant to possibly prevent disease recurrence and (3) to test the safety of combination Venetoclax and oral decitabine/cedazuridine as "maintenance therapy" after transplant to possibly prevent disease recurrence. * The name of the study drug involved in this study is Venetoclax. * It is expected that about 102 people will take part in this research study.

Timeline

Start

2018-10-24

Primary completion

2026-05-05

Completion

2030-03-05

Publications

• Background Garcia JS, Kim HT, Murdock HM, Cutler CS, Brock J, Gooptu M, Ho VT, Koreth J, Nikiforow S, Romee R, Shapiro R, Loschi F, Ryan J, Fell G, Karp HQ, Lucas F, Kim AS, Potter D, Mashaka T, Stone RM, DeAngelo DJ, Letai A, Lindsley RC, Soiffer RJ, Antin JH. Adding venetoclax to fludarabine/busulfan RIC transplant for high-risk MDS and AML is feasible, safe, and active. Blood Adv. 2021 Dec 28;5(24):5536-5545. doi: 10.1182/bloodadvances.2021005566.
• Background Garcia JS, Kim HT, Murdock HM, Ansuinelli M, Brock J, Cutler CS, Gooptu M, Ho VT, Koreth J, Nikiforow S, Romee R, Shapiro R, DeAngelo DJ, Stone RM, Bat-Erdene D, Ryan J, Contreras ME, Fell G, Letai A, Ritz J, Lindsley RC, Soiffer RJ, Antin JH. Prophylactic maintenance with venetoclax/azacitidine after reduced-intensity conditioning allogeneic transplant for high-risk MDS and AML. Blood Adv. 2024 Feb 27;8(4):978-990. doi: 10.1182/bloodadvances.2023012120.
• Garcia JS, Kim HT, Murdock HM, Bosch-Vilaseca A, Panaro KM, Lim F, Fiorilla J, Auriemma E, Brock J, Gooptu M, Ho VT, Cutler CS, Shapiro R, Kelkar AH, Abel GA, DeAngelo DJ, Stone RM, Bat-Erdene D, Ryan J, Fell G, Letai A, Ritz J, Lindsley RC, Antin JH, Soiffer RJ. Venetoclax/FluBu2 RIC transplant followed by all-oral venetoclax/decitabine maintenance for poor-risk MDS/AML. Blood Adv. 2026 Mar 10;10(5):1548-1558. doi: 10.1182/bloodadvances.2025018631.

Drugs

Indications