Study on Venetoclax and Chemotherapy Before Donor Stem Cell Transplant for Treating Acute Myelogenous Leukemia or Myelodysplastic Syndrome

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0

ClinicalTrials ID:NCT02250937

Provider View

This clinical trial is testing a combination of drugs, including venetoclax and chemotherapy, before a donor stem cell transplant to see if it can effectively treat patients with acute myelogenous leukemia or myelodysplastic syndrome.

Patient Parameters

Program Overview

The purpose of this study is to find out how well a combination of venetoclax and chemotherapy drugs (busulfan, cladribine, and fludarabine phosphate) works before a donor stem cell transplant in patients with acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS). The chemotherapy helps kill cancer cells and makes space in the bone marrow for new, healthy stem cells from a donor. These donor stem cells can help the patient's bone marrow produce healthy blood cells and may also help eliminate any remaining cancer cells.

Description

The study aims to compare two different schedules of giving venetoclax and chemotherapy before a stem cell transplant to see which is more effective in preventing the cancer from coming back. Researchers will also look at overall survival rates, side effects, and how well the new stem cells grow in the body. They will study the occurrence of graft-versus-host disease, where the donor cells attack the patient's body, and the rate of cancer relapse. Additionally, the study will explore how the cancer cells resist treatment, DNA damage, immune recovery, and specific markers in cancer cells.

Participants will be randomly assigned to one of two treatment groups:

  • Group 1: Patients will take venetoclax by mouth daily from day -22 to day -3. They will receive busulfan through an IV on days -13 and -12, followed by fludarabine phosphate, cladribine, and busulfan through an IV on days -6 to -3.

  • Group 2: Patients will take venetoclax by mouth daily from day -22 to day -3. They will receive busulfan through an IV on days -20 and -13, followed by fludarabine phosphate, cladribine, and busulfan through an IV on days -6 to -3.

After the chemotherapy, all patients will receive a donor stem cell transplant on day 0. Participants will be monitored for 2.5 years after the treatment to assess their health and the effectiveness of the treatment.

Eligibility Criteria

Inclusion Criteria

  • Diagnosed with acute myeloid leukemia or myelodysplastic syndrome, either with persistent disease or in remission.
  • Have a matching donor for the stem cell transplant, either a sibling or an unrelated donor.
  • Aged between 18 and 70 years old. Children aged 2-17 may join after safety is confirmed in adults.
  • Meet specific health criteria, including:
    • Direct bilirubin level below 1 mg/dl.
    • ALT level less than 3 times the upper limit of normal.
    • Creatinine clearance above 50 ml/min.
    • Lung function tests showing at least 50% of expected values.
    • Oxygen levels of at least 92% for young children.
    • Heart function with an ejection fraction of at least 40%.
  • Able to provide informed consent or assent for minors.
  • Women of childbearing potential must test negative for pregnancy and agree to use contraception.
  • Have a performance status score of 70 or higher, indicating good overall health.

Exclusion Criteria

  • Previous stem cell transplant, either from a donor or using their own cells.
  • Uncontrolled infections.
  • HIV positive.
  • High risk of complications from the transplant, as determined by a specific health score.
  • History of coronary artery disease.

Locations

FACILITYZIPCITYSTATE
M D Anderson Cancer Center77030HoustonTexas

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