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Study on the Safety and Effectiveness of Venetoclax with Fludarabine, Amsacrine, and Cytarabine for Patients with MDS, CMML, or Secondary AML Undergoing Stem Cell 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 evaluating the safety and effectiveness of a treatment plan for patients with certain types of blood disorders, specifically Myelodysplastic Syndrome (MDS), Chronic Myelomonocytic Leukemia (CMML), and Secondary Acute Myeloid Leukemia (sAML). The treatment involves the use of a medication called Venetoclax, which is taken orally in the form of a film-coated tablet. Venetoclax is being tested in combination with a series of other medications, including Fludarabine, Amsacrine, Ara-C (also known as Cytarabine), and Treosulfan, as part of a conditioning regimen before a procedure known as allogeneic blood stem cell transplantation.

The purpose of the study is to determine if adding Venetoclax to this treatment regimen is safe for patients undergoing the transplantation procedure. The study will follow participants through the course of their treatment, which includes taking the medications and undergoing the transplantation. The medications are administered in different ways: Venetoclax is taken orally, while Amsacrine and Cytarabine are given intravenously. The study will monitor the patients' health and any side effects they may experience during and after the treatment.

Participants in the study will be observed for a period of time after the transplantation to assess the safety of the treatment, including any potential organ toxicity and other adverse effects. The study will also look at how well the treatment works in terms of disease response and overall survival. This research aims to improve the outcomes for patients with high-risk forms of MDS, CMML, and sAML by finding the most effective and safe treatment combinations.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    Upon joining the trial, participation begins with an initial assessment to confirm eligibility based on specific medical conditions such as myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or secondary acute myeloid leukemia (sAML).

  2. Step 2

    Medication administration

    The trial involves the administration of several medications. Amsacrine is given intravenously, Mycophenolate mofetil is taken orally, Cytarabine is administered intravenously, Tacrolimus is taken orally, and Venetoclax is taken as a film-coated tablet orally.

    The specific dosages and frequency of these medications are determined by the trial protocol and are adjusted based on individual response and tolerance.

  3. Step 3

    Conditioning regimen

    The conditioning regimen includes a combination of medications such as Fludarabine, Amsacrine, and Ara-C (Cytarabine) along with Treosulfan. This regimen is designed to prepare the body for stem cell transplantation.

  4. Step 4

    Stem cell transplantation

    Following the conditioning regimen, an allogeneic blood stem cell transplantation is performed. This involves the transfer of healthy stem cells from a donor to replace the diseased or damaged bone marrow.

  5. Step 5

    Post-transplantation monitoring

    After transplantation, monitoring is conducted to assess safety and efficacy. This includes evaluating organ toxicity, adverse events, and the success of the graft.

    Monitoring continues for up to 100 days post-transplantation to observe disease response and donor chimerism, which indicates the presence of donor cells in the blood and marrow.

  6. Step 6

    Long-term follow-up

    Long-term follow-up is conducted to assess outcomes such as event-free survival, overall survival, and the incidence of complications like graft-versus-host disease (GvHD) and veno-occlusive disease (VOD).

    The trial aims to determine the maximum tolerated dose of Venetoclax and its safety when added to the conditioning regimen.

Who can join the trial?

7 criteria

  • The patient must have one of the following conditions: myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or secondary acute myeloid leukemia (sAML). These are specific types of blood disorders.
  • The patient must have a marrow blast count greater than 5%. This means that more than 5% of the cells in the bone marrow are immature blood cells.
  • The patient must have high-risk genetic features. This includes having certain genetic changes that are considered risky, such as specific mutations or changes in the DNA.
  • The patient must fall into the "high" or "very high" risk category according to specific medical guidelines used to assess the severity of the condition.
  • The patient must be between the ages of 18 and 64 years old.
  • Both male and female patients are eligible to participate.
  • The study includes patients who may be considered part of a vulnerable population, meaning they might need extra care or protection.

Who cannot join the trial?

9 criteria

  • Patients who have already received a stem cell transplantation. This is a procedure where healthy blood-forming cells are transferred to replace damaged or diseased bone marrow.
  • Patients who are not diagnosed with myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or secondary acute myeloid leukemia (sAML). These are specific types of blood disorders and cancers.
  • Patients who are not considered high-risk for the conditions mentioned above. High-risk means there is a greater chance of the disease progressing or not responding to treatment.
  • Patients who are not within the specified age range for the study. The study is open to certain age groups only.
  • Patients who are not able to safely undergo the treatment being tested in the study.
  • Patients who are not able to follow the study procedures or attend required visits.
  • Patients who have other medical conditions that might interfere with the study treatment or outcomes.
  • Patients who are pregnant or breastfeeding, as the study treatment may not be safe for the baby.
  • Patients who are participating in another clinical trial at the same time.
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Investigated drugs

  • Venetoclax

    is a medication used in this trial to help treat certain types of blood cancers. It works by targeting and blocking a specific protein that cancer cells need to survive, which may help to kill the cancer cells and slow down the progression of the disease.

  • Fludarabine

    is a chemotherapy drug used in this trial as part of the conditioning regimen before stem cell transplantation. It helps to suppress the immune system and destroy cancer cells, making it easier for the new stem cells to grow and develop in the patient's body.

  • Amsacrine

    is another chemotherapy medication included in the trial. It is used to kill cancer cells by interfering with their DNA, which prevents them from growing and dividing.

  • Ara-C

    , also known as Cytarabine, is a chemotherapy drug that is part of the treatment regimen in this trial. It works by stopping the growth of cancer cells, which helps to reduce the number of cancer cells in the body.

  • Treosulfan

    is a chemotherapy agent used in the trial to prepare the body for stem cell transplantation. It helps to eliminate cancer cells and suppress the immune system, allowing the transplanted stem cells to engraft and grow effectively.

What is already known about the treatment

Venetoclax – Venetoclax is administered orally in tablet form and is currently being studied in clinical trials for its safety and efficacy in combination with other drugs for patients with certain blood disorders. It is primarily used for patients with myelodysplastic syndrome, chronic myelomonocytic leukemia, or secondary acute myeloid leukemia before stem cell transplantation. Venetoclax works by inhibiting a protein called BCL-2, which helps cancer cells survive, thereby promoting their death. It is classified as a BCL-2 inhibitor and is part of targeted cancer therapies.

Investigated diseases

  • Myelodysplastic Syndrome (MDS)

    This is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the blood-forming cells in the bone marrow are damaged, leading to a shortage of one or more types of blood cells. Over time, MDS can progress to more severe forms of blood disorders, including acute myeloid leukemia. Patients may experience symptoms like fatigue, infections, or easy bruising due to low blood counts. The progression of MDS varies, with some cases remaining stable for years while others quickly advance to more serious conditions.

  • Chronic Myelomonocytic Leukemia (CMML)

    This is a type of cancer that starts in blood-forming cells of the bone marrow and invades the blood. It is characterized by an increase in monocytes, a type of white blood cell, and can lead to symptoms such as fatigue, fever, and weight loss. CMML can progress slowly or transform into acute myeloid leukemia. The disease often causes an enlarged spleen and liver due to the accumulation of abnormal cells. Over time, the bone marrow may become less effective at producing healthy blood cells.

  • Secondary Acute Myeloid Leukemia (sAML)

    This form of leukemia develops from a previous blood disorder or after exposure to certain chemotherapy treatments. 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 can include fatigue, frequent infections, and bleeding or bruising easily. sAML progresses quickly and requires prompt medical attention. The disease can lead to a significant reduction in the number of healthy blood cells, causing various health complications.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-514688-24-00Protocol codeFLAMSAClaxEstimated enrolment38 patientsSponsorHeinrich-Heine-Universitaet Duesseldorf

sourced from the EU Clinical Trials Register and site verification

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