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Study of Azacitidine and Drug Combination Before Stem Cell Transplantation in Patients with High-Risk Myelodysplastic Syndrome

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What is this trial about?

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

This study focuses on patients with Higher-risk Myelodysplastic Syndromes, a group of blood disorders where bone marrow does not produce enough healthy blood cells. The study evaluates two different treatment approaches: immediate hematopoietic stem cell transplantation (a procedure where damaged bone marrow is replaced with healthy stem cells) or transplantation after receiving other treatments.

The main treatment being studied is Azacitidine, which is given as an injection under the skin. Other medications that may be used in the study include Fludarabine, Daunorubicin, Busulfan, Cytarabine, and Thiotepa. These medications are given through an intravenous line directly into the bloodstream.

The purpose of this research is to determine whether it is better to perform stem cell transplantation immediately or to first treat patients with medications like Azacitidine or traditional chemotherapy, depending on the number of abnormal cells in their bone marrow. The study will monitor patients' health and recovery throughout their treatment period and follow-up care.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial evaluation

    A medical assessment will confirm if you have higher-risk myelodysplastic syndrome (a bone marrow disorder)

    Your bone marrow will be examined to determine the percentage of abnormal cells (blasts)

    Your overall health status and ability to perform daily activities will be evaluated

  2. Step 2

    Treatment assignment

    Based on your bone marrow blast percentage, you will be assigned to one of two treatment paths:

    If blast percentage is below 10%: direct stem cell transplantation

    If blast percentage is 10% or higher: pre-treatment before transplantation

  3. Step 3

    Pre-transplant treatment (if required)

    You may receive one of these treatments:

    Azacitidine - given by injection under the skin

    Conventional chemotherapy combination including: fludarabine, daunorubicin, cytarabine - given through intravenous infusion

  4. Step 4

    Stem cell transplantation

    Preparation with conditioning medications: busulfan and thiotepa given through intravenous infusion

    Stem cell transplantation procedure

    Hospital stay for monitoring and recovery

  5. Step 5

    Follow-up evaluations

    Bone marrow samples will be collected before transplant and 6 months after

    Quality of life assessments will be conducted

    Regular health monitoring will continue for several years

    Final study evaluation will be completed by September 2031

Who can join the trial?

7 criteria

  • Must be diagnosed with higher-risk myelodysplastic syndromes (HR-MDS) that has not been treated before - this is a blood disorder affecting bone marrow
  • Must be between 18 and 70 years old
  • Must be eligible for HSCT (stem cell transplantation - a procedure where damaged bone marrow is replaced with healthy cells)
  • Must have a life expectancy of at least 3 months
  • Must be able to perform daily activities with minimal assistance - specifically an ECOG Performance Status of 0-2 (a scale that measures a person's ability to perform daily activities and self-care)
  • Must be willing and able to provide written informed consent to participate in the study
  • Must not have received any previous treatment for HR-MDS

Who cannot join the trial?

14 criteria

  • Age below 18 years or above 65 years
  • Previous history of bone marrow transplant (a procedure where damaged bone marrow is replaced with healthy cells)
  • Active uncontrolled infections
  • Presence of more than 10% blast cells (immature blood cells) in bone marrow
  • Severe heart conditions including:
    • Uncontrolled heart failure
    • Recent heart attack (within last 6 months)
    • Unstable heart rhythm problems
    • Severe kidney or liver problems
    • Pregnancy or breastfeeding
    • Active other types of cancer
    • Mental conditions that prevent understanding of study requirements
    • Participation in other clinical trials within past 30 days
    • Known allergic reactions to study medications
    • Inability to follow study procedures
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Investigated drugs

  • Azacitidine

    is a medication used in treating myelodysplastic syndromes (MDS). It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. It's given as an injection under the skin or through an intravenous line.

  • Allogeneic stem cell transplantation

    is a procedure where healthy blood-forming stem cells from a matched donor are transferred to the patient. These cells help create new, healthy blood cells in the patient's body. This is not a medication but rather a therapeutic procedure that can potentially cure myelodysplastic syndromes.

  • Conventional chemotherapy

    refers to standard cancer-fighting drugs that are used to treat myelodysplastic syndromes. These medications work by killing rapidly dividing cells, including cancer cells. They are typically given through an intravenous line.

What is already known about the treatment

Azacitidine - A chemotherapy medication administered through subcutaneous injection or intravenous infusion, which is primarily used in the treatment of higher-risk myelodysplastic syndromes (HR-MDS). This DNA methyltransferase inhibitor works by preventing the methylation of DNA, which helps restore normal function to genes that control cell growth and differentiation in bone marrow cells. As a hypomethylating agent, it's considered a first-line treatment option for patients with HR-MDS, particularly before they undergo allogeneic stem cell transplantation, and has shown significant effectiveness in improving blood cell counts and reducing the need for blood transfusions.

Investigated diseases

Myelodysplastic Syndromes (Higher-risk) - A group of bone marrow disorders where the bone marrow doesn't produce enough healthy blood cells. In higher-risk forms, the bone marrow contains an increased number of immature blood cells called blasts. The condition affects the production of red blood cells, white blood cells, and platelets, leading to decreased blood cell counts. The bone marrow may produce blood cells that are abnormally shaped and don't function properly. This condition can gradually worsen over time as the number of abnormal cells increases and healthy cells decrease.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-510515-19-00Protocol codeMDS0519Estimated enrolment274 patientsSponsorFondazione Gimema Franco Mandelli Onlus

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