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New treatment option for Acute Myeloid Leukemia
Official title Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
ClinicalTrials.gov ID: NCT04708054
What this study is testing
What is Busulfan?
Busulfan is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for acute myeloid leukemia.
Also referred to as 1, 4-Bis[methanesulfonoxy]butane, BUS.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 70. Healthy volunteers may be eligible.
You may be able to join if
- Phase II
- Age ≥ 18 and ≤ 70 years. English and non-English speaking patients are eligible.
- Patients with acute myeloid leukemia who have previously received induction therapy and one of the following high-risk features:
- ELN17 adverse risk prognostic group irrespective of remission status (see Appendix 2)
- Measurable residual disease positive (MRD +)
You likely can't join if
- 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.
- Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
- Uncontrolled and/or active systemic infection (viral, bacterial or fungal)
See the full eligibility criteria
- Phase II
- Age ≥ 18 and ≤ 70 years. English and non-English speaking patients are eligible.
- Patients with acute myeloid leukemia who have previously received induction therapy and one of the following high-risk features:
- 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.
- AML secondary to MDS or MPD
- Therapy-related AML.
- Not in complete remission after one course of induction therapy Or Patients with myelodysplastic syndrome or CMML and one of the following high-risk features:
- Poor or Very poor cytogenetic risk group as per IPSS-R
- Mutated P53 or Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN1) or DNMT 3a or ASXL1 or RUNX1
- Maximum IPSS-R \>3.5 between diagnosis and the start of the preparative regimen.
- ≥ 5% BM blasts at transplant
- Therapy-related MDS
- HLA-identical sibling or a minimum of 7/8 matched unrelated donor, or a haploidentical related donor available
- Subject must voluntarily sign an informed consent
- Female people of childbearing potential must have negative results for pregnancy test
- Adequate hepatic and renal function per local laboratory reference range as follows:
- Aspartate transaminase (AST) and alanine transaminase (ALT) \< 3.0X ULN
- Bilirubin \<1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin)
- Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 50 mL/min; calculated by the Cockcroft Gault formula or measured by 24 hours urine collection. Phase III
- Age ≥ 18 and ≤ 65 years. English and non-English speaking patients are eligible.
- Patients with acute myeloid leukemia who have previously received induction therapy and one of the following high-risk features:
- ELN22 adverse risk prognostic group irrespective of remission status (see Appendix
- Measurable residual disease positive (MRD +) including MRD + any time after induction therapy.
- 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 4 for details.
- AML secondary to MDS or MPD
- Therapy-related AML.
- Not in complete remission after one course of induction therapy
- Second or higher complete remission Or Patients with myelodysplastic syndrome and one of the following high-risk features:
- Poor or Very poor cytogenetic risk group as per IPSS-R
- Mutated P53 or Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN11) or ASXL1 or RUNX1 or moderate high, or high, or very high-risk group as per IPSS-M
- Maximum IPSS-R \>3.5 between diagnosis and the start of the preparative regimen.
- ≥ 5% BM blasts at transplant
- Therapy-related MDS Or Patients with CMML
- HLA-identical sibling or a minimum of 7/8 matched unrelated donor
- Subject must voluntarily sign an informed consent
- Female people of childbearing potential must have negative results for pregnancy test
- Adequate hepatic and renal function per local laboratory reference range as follows:
- Aspartate transaminase (AST) and alanine transaminase (ALT) \< 3.0X ULN
- Bilirubin \<1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin)
- Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 50 mL/min; calculated by the Cockcroft Gault formula or measured by 24 hours urine collection.
- 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.
- Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
- Uncontrolled and/or active systemic infection (viral, bacterial or fungal)
- Chronic hepatitis B virus (HBV) or hepatitis C (HCV) requiring treatment. Note: people with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive...
- Cardiac history of CHF requiring treatment or Ejection Fraction \< 50% or unstable angina;
- Corrected DLCO \< 50% or FEV1 \<65%.
- Administration or consumption of any of the following within 3 days prior to the first dose of study drug:
- grapefruit or grapefruit products
- Seville oranges (including marmalade containing Seville oranges)
- star fruit
- Patients with cognitive impairments and/or any serious unstable pre-existing medical condition or psychiatric disorder that can interfere with safety or with obtaining informed consent or compliance with study...
- Prior allogeneic stem cell transplantation.
The study team makes the final eligibility decision.
Where it's taking place
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 70 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Houston, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.