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Study of venetoclax, treosulfan and fludarabine before stem cell transplant in patients with acute myeloid leukemia and myelodysplastic syndrome

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What is this trial about?

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

This clinical trial focuses on patients with Acute Myeloid Leukemia (AML), Myelodysplastic Neoplasm/Acute Myeloid Leukemia (MDS/AML), and higher risk Myelodysplastic Syndrome (MDS). These are blood and bone marrow disorders where abnormal blood cells are produced. The study uses a combination of three medications: Trecondi (treosulfan), Venclyxto (venetoclax), and Fludarabine, which work together to prepare patients for stem cell transplantation.

The purpose of this study is to evaluate the safety and feasibility of using this specific combination of medications before receiving stem cells from a donor (called allogeneic stem cell transplantation). The treatment involves receiving these medications through different methods - some are given as tablets by mouth, while others are administered through an intravenous line directly into the bloodstream.

During the study, patients will receive a carefully planned sequence of treatments over several days. Treosulfan will be given for three days, Fludarabine for five days, and Venetoclax for seven days. After this preparation phase, patients will receive stem cells from their matched donor. The doctors will then monitor the patients' progress and how well the transplanted cells are working in their body.

The research process

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

Step 1
Initial treatment phase
Treatment begins with three medications that work together to prepare your body for stem cell transplantation:
- Trecondi (treosulfan): administered through an intravenous infusion
- Venclyxto (venetoclax): taken as film-coated tablets by mouth
- Fludarabin: given through an intravenous infusion
Step 2
Stem cell transplantation
You will receive stem cells from a matched donor through your bloodstream
The transplantation occurs between day 14 and day 28 after the initial screening
The stem cells used will be Peripheral Blood Stem Cells (PBS)
Step 3
Post-transplant monitoring - Day 28
Medical evaluation to assess your overall survival and recovery
Checks for successful stem cell growth in your bone marrow (engraftment)
Step 4
Extended monitoring - Day 100
Comprehensive health assessment including:
- Bone marrow examination
- Blood count analysis
- Assessment of immune system recovery
- Quality of life evaluation
- Check for any signs of disease return
- Monitoring for transplant-related complications
Step 5
Long-term follow-up
Continued monitoring until the end of the study period (December 2027)
Regular health assessments and documentation of any medical events

Who can join the trial?

19 criteria

Age must be between 18 and 75 years when signing the consent form
Must have a suitable donor available
Must have adequate lung function with specific test results: - DLCO (ability of lungs to transfer oxygen) greater than 40% - FEV1/FVC (breathing capacity) greater than 50%
Heart function (LVEF) must be 40% or higher
Kidney function (GFR) must be at least 30 ml/min/1.73 m²
Liver function tests within specified limits: - Bilirubin not more than 3 times the upper normal limit - AST not more than 5 times the upper normal limit
Chest X-ray or CT scan must show no signs of active infection or other cancers
Must not have any active, uncontrolled infections
Must be willing to use effective birth control during treatment and for 6 months afterward
Women must have a negative pregnancy test (taken within 3 days of screening)
Must agree not to share medication with others
Must be fluent in German
Must be able to understand and sign informed consent
Must have confirmed diagnosis of AML, MDS/AML, or high-risk MDS (specific types of blood disorders)
Blood disorder must be under control at screening
Must be approved for stem cell transplant by a team of blood specialists
Must have a Karnofsky Performance Score (measure of physical ability) of 60% or higher
Must be scheduled for peripheral blood stem cell transplant
Stem cell transplant must be scheduled between 14 and 28 days after screening

Who cannot join the trial?

15 criteria

History of other cancers within 2 years before starting the study (except successfully treated non-melanoma skin cancer or cervical cancer)
Active, uncontrolled infections requiring intravenous (through the vein) antibiotics
Positive test for HIV (Human Immunodeficiency Virus)
Active hepatitis B or hepatitis C infection
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Severe heart conditions including:
- Heart failure with reduced heart function
- Unstable heart rhythm problems
- Heart attack within last 6 months
- Severe lung disease requiring oxygen therapy
- Severe kidney problems requiring dialysis (blood filtering treatment)
- Severe liver problems
- Pregnancy or breastfeeding
- Unable to provide informed consent
- Participation in other clinical trials within 30 days before this study
- Mental health conditions that could interfere with following study procedures
- Known allergic reactions to study medications

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Verified sites

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Universitaetsklinikum Tuebingen AöR
Tuebingen, Germany

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Investigated drugs

Venetoclax is a medication used to treat blood cancers. It works by blocking a protein that helps cancer cells survive, causing them to die. In this trial, it is used to enhance the conditioning treatment before stem cell transplantation. Treosulfan is a chemotherapy medication used in conditioning treatments before stem cell transplantation. It helps prepare the body for the transplant by reducing the number of existing blood cells and suppressing the immune system. This medication is given as part of a reduced intensity conditioning regimen, which means it's designed to be less harsh than traditional conditioning treatments. Allogeneic Stem Cell Transplantation is not a medication but a medical procedure where healthy blood-forming stem cells from a donor are transferred to the patient. This procedure is used to treat various blood disorders and blood cancers by replacing damaged or diseased bone marrow with healthy stem cells.

What is already known about the treatment

Venetoclax
A targeted oral medication used in the treatment of blood cancers, particularly Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS). This drug works by blocking the BCL-2 protein, which helps cancer cells survive, leading to their programmed cell death (apoptosis). Venetoclax belongs to the class of medications known as BCL-2 inhibitors and is typically administered in tablet form as part of combination therapy. The medication has shown significant promise in clinical trials for improving survival rates when used as part of conditioning regimens before stem cell transplantation.
Treosulfan
An alkylating agent used in conditioning regimens before stem cell transplantation, particularly in patients with blood disorders. It is administered intravenously and works by damaging the DNA of cancer cells, preventing them from dividing and ultimately causing their death. Treosulfan belongs to the alkylating agents class of chemotherapy drugs and is especially valuable in reduced-intensity conditioning protocols due to its favorable toxicity profile compared to traditional conditioning agents. The medication has demonstrated effectiveness in preparing patients with AML and MDS for stem cell transplantation while potentially reducing treatment-related complications.

Investigated diseases

Acute Myeloid Leukemia (AML)
A type of blood and bone marrow cancer that affects the production of normal blood cells. The disease develops when the bone marrow begins making abnormal white blood cells that grow and divide rapidly. These abnormal cells interfere with the production of normal blood cells, leading to a decrease in healthy blood cells. The condition progresses quickly if left untreated.
Myelodysplastic Syndrome (MDS)
A group of disorders where the bone marrow doesn't produce enough healthy blood cells. In this condition, immature blood cells fail to develop into healthy mature blood cells. The abnormal cells can build up in the bone marrow and blood. Over time, the lack of healthy blood cells can cause various health issues.
Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML)
A condition that occurs when myelodysplastic syndrome progresses and transforms into acute myeloid leukemia. This transformation happens when the number of immature blood cells (blasts) in the bone marrow increases significantly. The disease represents a continuum between MDS and AML.