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Venetoclax with Busulfan Regimen for High-Risk AML and MDS

This study is testing if adding venetoclax to a standard stem cell transplant regimen (busulfan, cladribine, and fludarabine) can improve outcomes for people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). These drugs work in different ways to stop cancer cells from growing. The study aims to see if this combination helps control these conditions better. You may be able to join if you are between 18 and 70 years old and have high-risk AML or MDS, including those with certain genetic markers (biomarkers) like KRAS, NF1, or NRAS. The main goal is to see how many participants are free from progression (worsening of the disease) one year after transplant. The current status of this study is unclear.

Study design
This is an interventional study with a planned enrollment of 324 participants. It has both a Phase 2 and a Phase 3 portion, with the Phase 3 comparing the experimental regimen to a standard of care.
What's involved
You would undergo a hematopoietic cell (stem cell) transplant and receive intravenous (IV) medications including busulfan, cladribine, fludarabine, and venetoclax.
Compensation
Not stated in the trial record.
Follow-up
The primary outcome is measured at one year post-transplant, suggesting follow-up for at least this duration.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04708054

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

Recruiting
PHASE2Ages 18–70InterventionalTreatment
M.D. Anderson Cancer Center
~324 participants
Updated 2026-08-11 on ClinicalTrials.gov
What's tested:BusulfanCladribineFludarabine PhosphateHematopoietic Cell TransplantationThiotepaVenetoclax

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
1-year progression free survival (PFS)
Measured over At 1 year post-transplant

Conditions

Acute Myeloid Leukemia
Chronic Myelomonocytic Leukemia
Myelodysplastic Syndrome

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Acute Myeloid LeukemiaChronic Myelomonocytic LeukemiaMyelodysplastic Syndrome

NCT04708054

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • M D Anderson Cancer Center

    Houston, Texasstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Uday R Popat · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

Uday R. Popat
[email protected]
713-745-3055
Email the study team

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Eligibility criteria

Inclusion

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.
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

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.

What this trial measures

  • 1-year progression free survival (PFS)At 1 year post-transplant

    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.