Not recruiting

Study of three different doses of busulfan with fludarabine and thymoglobuline in stem cell transplantation for patients with high-risk myeloid blood disorders

Verified siteRegistered drugNo placebo

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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 high-risk myeloid malignancies, which are serious blood disorders affecting bone marrow function. The research evaluates different treatment approaches for patients who will undergo stem cell transplantation from matched donors. The treatment combines three medications: Busulfan (given in different doses through intravenous infusion), Fludarabine, and Thymoglobuline.

The main purpose of this research is to determine how well patients respond to different doses of Busulfan when combined with the other medications as preparation therapy before stem cell transplantation. This preparation therapy, also known as conditioning therapy, helps prepare the body to receive the donor's stem cells.

During the study, patients will receive these medications through intravenous infusion. Busulfan will be given at different dose levels, while Fludarabine and Thymoglobuline will be administered at set doses. After receiving these medications, patients will undergo the stem cell transplantation procedure. The study will monitor how patients respond to the treatment over time, particularly focusing on whether the disease returns or progresses.

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 preparation
You will receive a combination of three medications through intravenous infusion (directly into your vein) as preparation for stem cell transplantation
This treatment is designed for patients with blood-related conditions that require stem cell transplantation from a matched donor
Step 2
Medication administration
The treatment involves three main medications:
- Busulfan (given as an infusion into your vein)
- Thymoglobuline (given as an infusion into your vein)
- Fludarabine (given as an infusion into your vein)
Step 3
Stem cell transplantation
After the preparation treatment, you will receive stem cells from your matched donor
The donor may be a family member or an unrelated person who matches your tissue type
Step 4
Recovery monitoring
Your blood cell counts will be monitored until they reach normal levels:
- White blood cells should reach 500 cells
- Platelets should reach 50,000 (without need for transfusions)
Your progress will be checked at 1 month, 2 months, and 3 months after transplantation
Step 5
Long-term follow-up
Your condition will be monitored for up to 2 years to track your progress
Regular check-ups will assess how well the treatment is working
Any side effects will be recorded and managed according to standard medical guidelines

Who can join the trial?

10 criteria

Patients with poor prognosis blood disorders, specifically:
- Myelodysplastic syndrome (a condition where blood cells don't develop properly)
- Acute Myeloid Leukemia (AML) in specific situations:
  - Beyond first complete remission, regardless of genetic features
  - After receiving two rounds of initial treatment
  - With no favorable risk factors according to established medical criteria
  - Patients must be either:
    
    Between 50 and 65 years old
    Under 50 years old but not suitable for stronger treatment methods involving radiation or combined chemotherapy
    Must have a matching donor who is either:
    
    A sibling with matching tissue type (HLA identical)
    An unrelated donor with complete tissue match (10/10 match)
    Must have valid social security coverage
    Must provide written informed consent to participate in the study

Who cannot join the trial?

15 criteria

Patients who have active, uncontrolled infections
Patients with severe heart problems or ejection fraction (heart's pumping ability) less than 40%
Patients with severe lung problems or diffusion capacity (ability to transfer oxygen) less than 50%
Patients with severe kidney problems or creatinine clearance (kidney function test) less than 50 ml/min
Patients with severe liver problems
Pregnant or breastfeeding women
Patients who are unable to provide informed consent
Patients with active psychiatric conditions that could interfere with treatment compliance
Patients with HIV infection (human immunodeficiency virus)
Patients with active hepatitis B or C infection
Patients who have participated in another clinical trial within the past 30 days
Patients with known allergies to study medications
Patients with uncontrolled high blood pressure
Patients who have had previous stem cell transplantation
Patients with other active cancers requiring treatment

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Assistance Publique Hopitaux De Paris
Paris, France
Verified
Besancon University Hospital Center
Besançon, France
Responsive
CHRU De Nancy
Vandoeuvre Les Nancy, France

Trial locations

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Investigated drugs

Busulfan (intravenous) is a chemotherapy medication used in stem cell transplantation. It helps prepare the body for transplantation by suppressing the immune system and making space in the bone marrow for new cells. Fludarabine is a chemotherapy drug that works by interfering with the growth of cancer cells and suppressing the immune system. It is commonly used in combination with other medications before stem cell transplantation. Thymoglobuline (also known as anti-thymocyte globulin) is an immune-suppressing medication that helps prevent rejection after transplantation. It works by reducing the activity of certain immune cells in the body. These medications are used together as a conditioning treatment before stem cell transplantation in patients with blood disorders affecting the myeloid cells (a type of blood cell).

What is already known about the treatment

Busulfan
An alkylating chemotherapy medication administered intravenously as part of conditioning therapy before stem cell transplantation. This drug works by damaging the DNA of cancer cells, preventing them from dividing and multiplying, and is particularly effective in treating myeloid malignancies. It belongs to the class of alkylating agents and is commonly used in combination with other medications like fludarabine for preparing patients for bone marrow transplantation.
Fludarabine
A chemotherapy medication given intravenously that belongs to the class of purine analogs, used in conditioning regimens before stem cell transplantation. It works by interfering with DNA synthesis in cancer cells, effectively stopping their growth and division. This drug is particularly effective in treating blood and bone marrow conditions when combined with other chemotherapy agents like busulfan.
Thymoglobuline
An immunosuppressive medication administered intravenously that contains antibodies targeting specific immune cells. It is used in transplant procedures to prevent rejection and graft-versus-host disease by reducing the activity of T-lymphocytes in the immune system. This medication helps prepare the patient's body for stem cell transplantation by modifying the immune response.

Investigated diseases

Myeloid Malignancies
A group of blood and bone marrow disorders where abnormal blood cells develop from myeloid stem cells. These conditions affect the production and function of blood cells, including red blood cells, white blood cells, and platelets. The disease progresses when abnormal cells multiply and crowd out healthy blood cells in the bone marrow. This condition can develop gradually or rapidly, depending on the specific type of myeloid malignancy. Over time, the bone marrow's ability to produce normal blood cells becomes increasingly impaired.
Graft Versus Host Disease (GVHD)
A condition that occurs after receiving cells from a donor, where the transplanted cells recognize the recipient's body as foreign. The condition can affect various organs and tissues, including the skin, liver, and digestive tract. GVHD can develop within weeks of transplantation (acute) or months later (chronic). The condition causes various symptoms depending on which organs are affected.