Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18-70
SponsorM.D. Anderson Cancer Center
About this trial
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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
Age ≥ 18 and ≤ 70 years. English and non-English speaking patients are eligible.
ELN17 adverse risk prognostic group irrespective of remission status (see Appendix 2)
Measurable residual disease positive (MRD +)
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.
Disqualifiers
Subject is known to be positive for HIV.
Subject has cognitive impairments and/or is a prisoner.
Subject has acute promyelocytic leukemia
Subject has known active CNS involvement with AML.
Trial design
Design model
Single group
Treatments tested in this trial
Busulfan
DrugGiven IV
Cladribine
DrugGiven IV
Fludarabine Phosphate
DrugGiven IV
Hematopoietic Cell Transplantation
Procedure/SurgeryUndergo stem cell transplantation
Thiotepa
DrugGiven IV
Venetoclax
DrugGiven PO
Treatment groups
324 Participants
are divided into 1 treatment groupGroup A: Treatment (venetoclax, busulfan, fludarabine, cladribine)Experimental treatment 6 interventions
Trial outcomes
Primary outcomes
1
1-year progression free survival (PFS)
The proportion of patients who are alive without disease relapse (PFS) at one year will be reported along with the corresponding 95% credible interval. Cox proportional hazards regression will be used to assess the association between PFS and clinical and treatment covariates of interest.
Time frame
At 1 year post-transplantSecondary outcomes
1
Overall survival (OS)
OS will be calculated from the time of transplant by the method of Kaplan and Meier.
Time frame
Up to 3 years post-transplant2
Graft-versus (vs.)-host disease-free, relapse-free survival (GRFS)
GRFS will be calculated from the time of transplant by the method of Kaplan and Meier.
Time frame
Up to 3 years post-transplant3
Time to platelet engraftment
The time to platelet engraftment will be calculated from the time of transplant and estimated by the Kaplan-Meier method.
Time frame
From the time of transplant up to 3 years4
Time to neutrophil engraftment
The time to neutrophil engraftment will be calculated from the time of transplant and estimated by the Kaplan-Meier method.
Time frame
From the time of transplant up to 3 yearsSponsors and contacts
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Source ClinicalTrials.gov