Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
M.D. Anderson Cancer Center
Summary
This phase II trial studies the effect of venetoclax together with busulfan, cladribine, and fludarabine in treating patients with high-risk acute myeloid leukemia or myelodysplastic syndrome who are undergoing stem cell transplant. Chemotherapy drugs, such as venetoclax, busulfan, cladribine, and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding venetoclax to the current standard of care stem cell transplant regimen of busulfan, fludarabine, and cladribine may help to control high-risk acute myeloid leukemia or myelodysplastic syndrome.
Description
Phase 2 Portion Primary Objective 1) To obtain preliminary evidence of efficacy as defined by 1-year progression free survival. Secondary Objectives To determine: 1. Safety of this regimen as per NCI toxicity criteria 2. Time to neutrophil and platelet engraftment 3. Incidence of acute and chronic GVHD 4. Relapse incidence 5. Non-relapse mortality 6. Overall survival 7. Graft versus host disease-relapse free survival (GRFS) Phase 3 Portion Primary Objective 1\) To compare progression free survival between two arms Arm 1 Standard of Care: fludarabine + IV busulfan (FluBu) versus Arm 2 Ex…
Eligibility
- Age range
- 18–70 years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: Phase II 1. Age ≥ 18 and ≤ 70 years. English and non-English speaking patients are eligible. 2. Patients with acute myeloid leukemia who have previously received induction therapy and one of the following high-risk features: 1. ELN17 adverse risk prognostic group irrespective of remission status (see Appendix 2) 2. Measurable residual disease positive (MRD +) 3. Not in complete remission including complete remission without count recovery (Cri) and/or morphologic leukemia free state (MLFS), primary refractory, or relapsed disease. See Appendix 3 for details.…
Interventions
- DrugBusulfan
Given IV
- DrugCladribine
Given IV
- DrugFludarabine Phosphate
Given IV
- ProcedureHematopoietic Cell Transplantation
Undergo stem cell transplantation
- DrugThiotepa
Given IV
- DrugVenetoclax
Given PO
Location
- M D Anderson Cancer CenterHouston, Texas