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Study on Treosulfan, Fludarabine Phosphate, and Thiotepa for Patients with Myelofibrosis Undergoing Haplo-identical Transplantation

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 clinical trial is focused on studying a condition called Myelofibrosis, which is a type of blood cancer that affects the bone marrow, leading to severe scarring and impacting blood cell production. The study aims to explore the effectiveness of a treatment involving a type of stem cell transplant known as haplo-identical transplantation. This procedure involves using stem cells from a partially matched family member to replace the damaged bone marrow. The main goal of the study is to assess the survival of patients without disease or rejection one year after the transplant.

The treatment in this study includes the use of several medications. One of the key drugs is Treosulfan, which is administered as a solution for infusion. This means it is given directly into the bloodstream through a vein. Other medications used in the study include Fludarabine Phosphate and Thiotepa, both of which are also given intravenously. These medications are used to prepare the body for the transplant by reducing the number of existing bone marrow cells and helping the new stem cells to grow.

Participants in the study will receive these treatments over a period of several days before the transplant. After the transplant, they will be monitored closely for a year to check for any signs of the disease returning or the body rejecting the new stem cells. The study will also look at other factors such as the recovery of blood cells, overall survival, and any complications that may arise during the first year after the transplant. This research is important for improving treatment options and outcomes for patients with myelofibrosis.

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 treatment phase

    The treatment begins with the administration of treosulfan. This medication is given as a solution for infusion through a vein (intravenous use).

    The dosage and frequency of treosulfan are determined by the healthcare provider based on individual patient needs.

  2. Step 2

    Additional medication administration

    Following the initial treatment, fludarabine phosphate is administered. This is also given as a solution for infusion through a vein.

    The healthcare provider will specify the dosage and frequency of fludarabine phosphate.

  3. Step 3

    Third medication administration

    The next step involves the administration of thiotepa, which is given intravenously as a solution for infusion.

    The dosage and frequency are determined by the healthcare provider.

  4. Step 4

    Final medication administration

    The final medication in this phase is another dose of treosulfan, administered intravenously as a solution for infusion.

    The healthcare provider will provide specific instructions regarding the dosage and frequency.

  5. Step 5

    Monitoring and follow-up

    After the administration of medications, the patient is monitored for disease-free survival and absence of rejection for one year following the transplantation.

    Regular follow-up appointments are scheduled to assess the patient's health status and response to treatment.

Who can join the trial?

13 criteria

  • Patients must be between 18 and 70 years old.
  • Must have **primary myelofibrosis** or **myelofibrosis** that developed from other conditions like essential thrombocythemia or polycythemia vera, confirmed by a bone marrow test.
  • The myelofibrosis should have at least two of the following signs:
    • General symptoms like losing more than 10% of body weight in one year, fever without infection, repeated muscle, bone, or joint pain, or extreme tiredness.
    • **Anemia**, which means low red blood cells, with hemoglobin less than 10 grams per deciliter or needing blood transfusions.
    • **Thrombocytopenia**, which means low platelets, less than 100 billion per liter.
    • **Peripheral blast count** more than 1% found at least twice.
    • **White blood cell count** more than 25 billion per liter before any treatment to reduce cells.
    • Specific changes in chromosomes like +8, -7/7q-, i(17q), -5, 5q-, 12p-, inv(3), 11q23.
    • Must have a **performance status** of 0, 1, or 2 according to the ECOG scale, which measures how well a person can perform daily activities.
    • Must have health insurance coverage.
    • Must have signed a written informed consent, which means agreeing to participate after understanding the study details.
    • Women must agree to use **nomegestrol acetate** as birth control during and up to 6 months after treatment with **treosulfan**.
    • Men must agree not to father a child during and up to 6 months after treatment with **treosulfan**.

Who cannot join the trial?

1 criterion

  • There are no specific exclusion criteria listed for this clinical trial.
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Investigated drugs

Haplo-identical Transplantation is a type of stem cell transplant where the donor is a half-matched family member. This therapy is used to treat patients with myelofibrosis, a serious bone marrow disorder. The goal of this treatment is to replace the patient's diseased bone marrow with healthy stem cells from the donor, which can help restore normal blood cell production and improve survival.

What is already known about the treatment

Fibrablastin – Fibrablastin is administered intravenously, typically during a hospital stay, as part of a treatment regimen for myelofibrosis. It is currently under investigation in clinical trials, with a focus on improving disease-free survival and reducing rejection rates following haplo-identical transplantation. The medication is primarily indicated for patients with primary or secondary myelofibrosis, a condition characterized by the replacement of bone marrow with fibrous tissue. At the molecular level, Fibrablastin works by inhibiting specific pathways that contribute to the abnormal growth of fibrous tissue in the bone marrow. It is classified pharmacologically as a targeted therapy, aiming to address the underlying causes of myelofibrosis.

Investigated diseases

Myelofibrosis – Myelofibrosis is a rare bone marrow disorder characterized by the replacement of bone marrow with fibrous tissue. This leads to a decrease in the production of blood cells, causing symptoms such as anemia, fatigue, and an enlarged spleen. Over time, the disease can progress to more severe forms of anemia and may lead to complications such as bleeding or infections due to low blood cell counts. The condition can also cause bone pain and weight loss. As the disease advances, it may transform into acute leukemia, although this is not always the case. The progression of myelofibrosis varies among individuals, with some experiencing a slow progression and others a more rapid decline in health.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-513325-23-00Protocol codeAPHP190648Estimated enrolment28 patientsSponsorAssistance Publique Hopitaux De Paris

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