RecruitingPhase 2 / Phase 3

Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 70 Years
Sex
All sexes
Healthy volunteers
Accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests adding venetoclax to busulfan, cladribine, and fludarabine before a stem cell transplant. It is for adults with high-risk acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelomonocytic leukemia (CMML), which affect blood and bone marrow. Researchers want to see if adding venetoclax helps control the disease.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 18–70 for Phase II or 18–65 for Phase III. You do not need to speak English.
  • For AML: have had initial treatment and meet the study’s high-risk rules. These include certain gene changes, cancer cells left after treatment, disease that returned, or AML linked to an earlier blood disease or cancer treatment.
  • For MDS: meet the study’s high-risk rules. These include certain gene changes, high risk scores, at least 5% immature cells in bone marrow, or MDS linked to earlier cancer treatment.
  • For CMML: meet the listed high-risk rules for Phase II. Phase III lists CMML without added high-risk rules.
  • Have a fully matched sibling donor or an unrelated donor matching at least 7 of 8 tested markers. Phase II also allows a half-matched related donor.
  • Choose to join and sign a consent form after learning about the study.
  • If you can become pregnant, have a negative pregnancy test.
  • Meet the study’s liver and kidney test limits. Some causes of a high bilirubin level, a liver blood test, are allowed.

Who can't join

  • Have HIV, or hepatitis B or C that needs treatment. Signs of past hepatitis B vaccination or certain antibodies from IVIG treatment do not rule you out.
  • Have problems with thinking or understanding, or be a prisoner.
  • Have acute promyelocytic leukemia, a type of AML, or active AML in the brain or spinal cord.
  • Have an uncontrolled serious health problem, an active infection affecting the body, or an unstable medical or mental health condition that could affect safe participation.
  • Have heart failure needing treatment, a heart pumping measure below 50%, or unstable chest pain.
  • Have lung test results below the study’s limits: corrected DLCO below 50% or FEV1 below 65%. These tests measure breathing and oxygen transfer.
  • Eat grapefruit products, Seville oranges or marmalade made with them, or star fruit within 3 days before the first study drug dose.
  • Have already had a stem cell transplant using cells from another person.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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. 4. AML secondary to MDS or MPD 5. Therapy-related AML. 6. 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: 1. Poor or Very poor cytogenetic risk group as per IPSS-R 2. Mutated P53 or Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN1) or DNMT 3a or ASXL1 or RUNX1 3. Maximum IPSS-R \>3.5 between diagnosis and the start of the preparative regimen. 4. ≥ 5% BM blasts at transplant 5. Therapy-related MDS 3. HLA-identical sibling or a minimum of 7/8 matched unrelated donor, or a haploidentical related donor available 4. Subject must voluntarily sign an informed consent 5. Female subjects of childbearing potential must have negative results for pregnancy test 6. 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 1. Age ≥ 18 and ≤ 65 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. ELN22 adverse risk prognostic group irrespective of remission status (see Appendix 2. Measurable residual disease positive (MRD +) including MRD + any time after induction therapy. 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 4 for details. 4. AML secondary to MDS or MPD 5. Therapy-related AML. 6. Not in complete remission after one course of induction therapy 7. Second or higher complete remission Or Patients with myelodysplastic syndrome and one of the following high-risk features: 1. Poor or Very poor cytogenetic risk group as per IPSS-R 2. 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 3. Maximum IPSS-R \>3.5 between diagnosis and the start of the preparative regimen. 4. ≥ 5% BM blasts at transplant 5. Therapy-related MDS Or Patients with CMML 3. HLA-identical sibling or a minimum of 7/8 matched unrelated donor 4. Subject must voluntarily sign an informed consent 5. Female subjects of childbearing potential must have negative results for pregnancy test 6. 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. Exclusion criteria: 1. Subject is known to be positive for HIV. 2. Subject has cognitive impairments and/or is a prisoner. 3. Subject has acute promyelocytic leukemia 4. Subject has known active CNS involvement with AML. 5. Evidence of other clinically significant uncontrolled condition(s) including, but not limited to: 1. Uncontrolled and/or active systemic infection (viral, bacterial or fungal) 2. Chronic hepatitis B virus (HBV) or hepatitis C (HCV) requiring treatment. Note: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive and anti-hepatitis B core (HBc) antibody negative) or positive anti-HBc antibody from intravenous immunoglobulins (IVIG) may participate. 6. Cardiac history of CHF requiring treatment or Ejection Fraction \< 50% or unstable angina; 7. Corrected DLCO \< 50% or FEV1 \<65%. 8. 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 9. 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 procedures. 10. Prior allogeneic stem cell transplantation.

Study sites (1)

TX