Actively Recruiting

Phase 2
Phase 3
Age: 18Years - 70Years
All Genders
Healthy Volunteers
NCT04708054

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

Led by M.D. Anderson Cancer Center · Updated on 2026-02-19

324

Participants Needed

1

Research Sites

323 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years - 70Years
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 to 70 years for Phase II; 18 to 65 years for Phase III
  • Diagnosed with acute myeloid leukemia with high-risk features such as ELN adverse risk or measurable residual disease positive
  • Diagnosed with myelodysplastic syndrome or chronic myelomonocytic leukemia with poor cytogenetic risk or specific gene mutations
  • Available HLA-identical sibling, 7/8 matched unrelated donor, or haploidentical related donor
  • Voluntarily signed informed consent
  • Female participants of childbearing potential must have a negative pregnancy test
  • Adequate liver function: AST and ALT less than 3 times the upper limit of normal, bilirubin less than 1.5 times the upper limit (unless due to non-hepatic causes)
  • Adequate kidney function with creatinine clearance of at least 50 mL/min
Not Eligible

You will not qualify if you...

  • Known HIV positive status
  • Cognitive impairments or prisoner status
  • Diagnosis of acute promyelocytic leukemia
  • Active central nervous system involvement with AML
  • Uncontrolled or active infections (viral, bacterial, or fungal)
  • Chronic hepatitis B or C requiring treatment (except certain prior vaccination or antibody positive cases)
  • History of congestive heart failure requiring treatment or ejection fraction less than 50%, or unstable angina
  • Lung function with corrected DLCO less than 50% or FEV1 less than 65%
  • Consumption of grapefruit, Seville oranges, or star fruit within 3 days prior to first study dose
  • Serious unstable medical or psychiatric conditions interfering with safety or informed consent
  • Prior allogeneic stem cell transplantation

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

U

Uday R. Popat

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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