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Study Comparing Treosulfan and Melphalan with Fludarabine for Patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS) Undergoing Transplantation

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying two blood-related diseases: Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS). These conditions affect the blood and bone marrow, leading to issues with blood cell production. The study involves patients who are undergoing a type of treatment called allogeneic transplantation, where they receive healthy stem cells from a donor to help restore their bone marrow function.

The purpose of the study is to evaluate the effectiveness of a combination of medications used during the transplantation process. The medications being studied are Fludarabine, Treosulfan, and Melphalan. Fludarabine is used in combination with either Treosulfan or Melphalan to prepare the body for the transplant. These medications are given through an intravenous infusion, which means they are delivered directly into the bloodstream through a vein. The study will compare the outcomes of using Treosulfan versus Melphalan in combination with Fludarabine to see which is more effective in helping patients with AML and MDS.

Participants in the study will receive one of the two treatment combinations and will be monitored for their response to the treatment. The study will also look at the prevention of a condition called Graft-versus-Host Disease (GvHD), which can occur after a transplant when the donor cells attack the recipient's body. The study aims to find the best treatment approach to improve survival rates and reduce complications for patients undergoing allogeneic transplantation for AML and MDS.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment phase

    The treatment begins with the administration of fludarabine, melphalan, or treosulfan. These medications are given through an intravenous infusion, which means they are delivered directly into the bloodstream through a vein.

    The specific medication used will depend on the randomization process of the trial. The purpose of these medications is to prepare the body for the transplantation process.

  2. Step 2

    Transplantation phase

    Following the initial treatment, the patient will undergo an allogeneic transplantation. This involves receiving healthy stem cells from a donor to replace the patient's damaged or diseased bone marrow.

    The transplantation is a critical step in the treatment process and aims to restore the body's ability to produce healthy blood cells.

  3. Step 3

    Post-transplantation care

    After the transplantation, the patient will receive post-transplantation care, which includes PTCy-based GvHD prophylaxis. This involves the administration of a medication called cyclophosphamide to prevent graft-versus-host disease (GvHD), a condition where the donor cells attack the patient's body.

    The prophylaxis is given on days +3 and +4 after the transplantation, with a dosage of 50 mg per kg of body weight.

  4. Step 4

    Monitoring and follow-up

    The patient will be closely monitored for overall survival and any potential side effects or complications. This includes regular check-ups and tests to assess the success of the transplantation and the patient's recovery.

    The monitoring will also focus on the incidence of relapse, the rate of engraftment, and the development of any acute or chronic GvHD.

Who can join the trial?

21 criteria

  • The patient must sign an informed consent form, which is a document that explains the study and confirms the patient's willingness to participate.
  • Men must agree to avoid unprotected sex and not donate sperm from the time they sign the consent form until 6 months after their last dose of the study drug.
  • Women must meet at least one of the following conditions:
    • Be post-menopausal, meaning they haven't had a menstrual period for 12 months naturally or 6 months with a specific hormone level.
    • Have had surgery to remove both ovaries, with or without the uterus, at least 6 weeks ago.
    • If they can still have children, they must have a negative pregnancy test within 7 days before the first dose of the study drug.
    • Use a reliable birth control method from the time they sign the consent form until 6 months after their last dose of the study drug.
    • Choose not to have sex from the time they sign the consent form until 6 months after their last dose of the study drug.
    • Have a partner who has had a vasectomy, which is a procedure to prevent sperm from being part of the semen.
    • The patient must be scheduled for an allogeneic transplantation, which is a type of stem cell transplant, within the next 3 weeks.
    • The patient must be 18 years of age or older.
    • The patient must have either **AML** (Acute Myeloid Leukemia) or **MDS** (Myelodysplastic Syndromes) as defined by the **WHO** (World Health Organization) and need an allogeneic **HCT** (Hematopoietic Cell Transplantation).
    • The patient must have an increased risk for treatment-related side effects from a strong conditioning treatment, meeting at least one of these criteria:
      • Be 50 years or older at the time of transplant.
      • Have a **HCT-CI** (Hematopoietic Cell Transplantation-Comorbidity Index) score greater than 2.
      • Be scheduled for a second allogeneic HCT from a different donor, with at least 12 months since the first transplant.
      • The patient must have a suitable donor, which can be:
        • A matched sibling donor.
        • A matched unrelated donor with a perfect match (10/10) in specific genetic markers called **HLA** (Human Leukocyte Antigen).
        • A mismatched unrelated donor with a single mismatch in specific HLA markers but no mismatch in another marker called **DQB1** (9/10 match).
        • A haploidentical family donor, which means a family member who is a half-match.
        • The patient must have a planned **GvHD** (Graft-versus-Host Disease) prevention treatment using standard **PTCy** (Post-Transplant Cyclophosphamide) on specific days after the transplant.
        • The patient must not have a history of heart disease that would prevent the transplant and must not have active heart symptoms. If there are heart concerns, the heart's pumping ability, called the left ventricular ejection fraction, must be 40% or higher.
        • The patient must not need extra oxygen on the day they are randomly assigned to a treatment group in the study.

Who cannot join the trial?

5 criteria

  • Patients who are not diagnosed with AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) and are not undergoing an allogeneic transplantation cannot participate.
  • Patients who are not in the 1st or 2nd CR (Complete Remission) for AML or do not have MDS are excluded.
  • Individuals who do not fall within the specified age ranges for the study are not eligible.
  • Both male and female patients are eligible, but those who do not meet other criteria will be excluded.
  • Patients who are considered part of a vulnerable population, which may include those unable to give informed consent, may be excluded.
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Investigated drugs

  • Fludarabine

    is a medication used to help prepare the body for a stem cell transplant. It works by suppressing the immune system, which helps the body accept the new stem cells.

  • Treosulfan

    is used as part of the conditioning regimen before a stem cell transplant. It helps to destroy any remaining cancer cells and makes space in the bone marrow for the new stem cells to grow.

  • Melphalan

    is another medication used in the conditioning process before a stem cell transplant. It helps to eliminate cancer cells and prepare the bone marrow for the new stem cells.

  • PTCy (Post-Transplant Cyclophosphamide)

    is used after a stem cell transplant to prevent graft-versus-host disease (GvHD), a condition where the new stem cells attack the patient's body. It helps to protect the patient from this complication by suppressing the immune response.

What is already known about the treatment

  • Treosulfan

    Treosulfan is administered intravenously and is currently being evaluated in clinical trials for its effectiveness in conditioning regimens for patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) undergoing allogeneic transplantation. It is primarily used as a conditioning agent before stem cell transplantation. Treosulfan works by interfering with the DNA of cancer cells, preventing them from dividing and growing. It is classified as an alkylating agent, which is a type of chemotherapy drug.

  • Melphalan

    Melphalan is given intravenously and is also under investigation in clinical trials for its role in conditioning regimens for patients with AML and MDS undergoing allogeneic transplantation. It is used to prepare the body for stem cell transplantation by destroying cancer cells and suppressing the immune system. Melphalan acts by binding to DNA and disrupting the replication of cancer cells. It belongs to the class of alkylating agents, which are commonly used in chemotherapy.

Investigated diseases

  • Acute Myeloid Leukemia (AML)

    A type of cancer that starts in the blood-forming cells of the bone marrow and quickly progresses to affect the blood. It is characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. Symptoms may include fatigue, fever, frequent infections, and easy bruising or bleeding. As the disease progresses, it can spread to other parts of the body, including the lymph nodes, liver, and spleen. AML is more common in adults and can vary in its aggressiveness and response to treatment.

  • Myelodysplastic Syndromes (MDS)

    A group of disorders caused by poorly formed or dysfunctional blood cells due to abnormal development in the bone marrow. These syndromes can lead to anemia, infections, or bleeding due to the lack of healthy blood cells. MDS can remain stable for years or progress to more severe forms of blood cancer, such as acute myeloid leukemia. Symptoms often include fatigue, shortness of breath, and increased susceptibility to infections. The condition is more common in older adults and can vary widely in severity and progression.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-507879-21-00Protocol codeTUD-ETAL-5-084Estimated enrolment220 patientsSponsorTechnische Universitaet Dresden

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