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Testing Stem Cell Transplant Methods and Medications Including Ruxolitinib and Blinatumomab for Children and Young Adults with Acute Lymphoblastic Leukemia

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What is this trial about?

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

This study involves patients with acute lymphoblastic leukemia, which is a type of blood cancer that affects white blood cells. The study focuses on children, teenagers, and young adults up to 25 years of age who need a stem cell transplant, which is a procedure where diseased bone marrow is replaced with healthy stem cells from a donor. The study will use several different medications including ruxolitinib, which is taken by mouth as a tablet or liquid, blinatumomab, which is given through a vein, and various chemotherapy medicines such as treosulfan, busulfan, fludarabine, cyclophosphamide, etoposide, and thiotepa. Some patients will also receive medicines to help prevent complications after transplant, including anti-t lymphocyte immunoglobulin and methylprednisolone, which is a type of steroid medicine. Some patients in the study may receive a lower dose of radiation treatment called total body irradiation before their transplant.

The purpose of this study is to find better ways to perform stem cell transplants in young patients with acute lymphoblastic leukemia by reducing side effects and complications while still keeping the transplant effective at preventing the cancer from coming back. The study will test different approaches, including whether a lower dose of radiation works as well as a higher dose, whether adding ruxolitinib to steroids helps treat a complication called graft-versus-host disease, which happens when the donor cells attack the patient's body, and whether giving blinatumomab after transplant helps prevent the cancer from returning in very young children. The study will also compare different types of donors and different ways to prepare patients for transplant.

Patients will be followed for several years after their transplant to see how well the treatments work. During the study, patients will receive their transplant preparation treatment over several days, followed by the stem cell transplant itself. Some patients may receive additional treatment after transplant depending on which part of the study they are in. For example, patients receiving ruxolitinib for graft-versus-host disease may take this medicine for up to 24 weeks, while very young children may receive up to four cycles of blinatumomab treatment after their transplant. Throughout the study, patients will have regular check-ups and tests to monitor their progress and watch for any side effects or complications.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Confirmation of eligibility and enrollment

    Your eligibility for the trial will be confirmed. This includes verification that you are between 3 months and 25 years of age at the time of transplant.

    You must be in complete remission, which means less than 5% abnormal cells in your bone marrow and no signs of disease in other parts of your body.

    If you are female and able to become pregnant, a pregnancy test will be performed, which must be negative.

    You will be assigned to one or more specific parts of the trial based on your age, donor type, and other medical factors.

  2. Step 2

    Preparation for transplant - conditioning regimen

    You will receive a conditioning regimen, which is a combination of medications given before the transplant to prepare your body. The specific medications and doses depend on which part of the trial you are enrolled in.

    The conditioning may include total body irradiation (radiation treatment to the whole body) at different dose levels, either 8 Gray or 12 Gray, combined with other medications.

    Medications used in conditioning may include: busulfan (given through a vein), etoposide (given through a vein), cyclophosphamide (given through a vein), fludarabine (given through a vein), thiotepa (given through a vein), or treosulfan (given through a vein).

    These medications work to eliminate any remaining disease cells and suppress your immune system so your body will accept the transplanted cells.

  3. Step 3

    Stem cell transplant procedure

    You will receive the stem cell transplant, also called hematopoietic stem cell transplant or HSCT. This involves receiving healthy blood-forming cells from a donor through a vein.

    The donor may be a matched sibling, a matched unrelated donor, or a mismatched family donor, depending on availability and your specific situation.

    The transplant may use cells collected from bone marrow, peripheral blood, or cord blood.

  4. Step 4

    Prevention of graft-versus-host disease

    You will receive medications to prevent graft-versus-host disease, a condition where the transplanted cells attack your body. The specific approach depends on your donor type.

    Preventive medications may include anti-t lymphocyte immunoglobulin (given through a vein), which helps prevent your body from rejecting the transplant.

    If you receive cells from a mismatched family donor, prevention may involve either post-transplant cyclophosphamide (given through a vein after the transplant) or removal of specific cell types from the donor cells before transplant.

    Additional medications such as methylprednisolone (a steroid given through a vein) may be used.

  5. Step 5

    Treatment if graft-versus-host disease develops (if applicable)

    If you are between 3 months and 18 years old, received cells from a matched donor, and develop acute graft-versus-host disease (grades II to IV), you may be assigned to receive additional treatment.

    You will receive either corticosteroids (anti-inflammatory medications) alone or corticosteroids combined with ruxolitinib.

    If assigned to receive ruxolitinib, it will be given as tablets or oral solution by mouth. The treatment response will be evaluated at day 28 after starting this therapy.

  6. Step 6

    Maintenance therapy after transplant (if applicable)

    If you are younger than 2 years old at the time of transplant and your disease cells expressed a marker called CD19, you may receive maintenance therapy.

    This maintenance therapy consists of up to four cycles of blinatumomab, a medication given through continuous infusion into a vein.

    This treatment is designed to reduce the risk of disease returning after transplant.

    You must be in complete remission and have no signs of disease in your brain or spinal fluid at the time this treatment begins.

  7. Step 7

    Monitoring and follow-up

    You will be monitored regularly for signs of disease relapse, transplant complications, and overall health.

    Blood tests will be performed to assess various factors, including a measurement called EASIX, which helps predict transplant-related complications.

    If your donor has a different blood type than yours (called ABO major incompatibility), special monitoring will be performed according to standard procedures at the treatment center.

    Follow-up will continue for several years after transplant, with specific assessments at 2 years and 4 years to evaluate treatment outcomes.

Who can join the trial?

9 criteria

  • The patient must have a diagnosis of ALL, which stands for acute lymphoblastic leukemia, a type of blood cancer
  • The patient must be between 3 months and 25 years old at the time of the transplant, which is a procedure where healthy blood-forming cells are given to replace damaged ones
  • The patient must be in complete remission, meaning there are fewer than 5 out of 100 abnormal cells in the bone marrow and no cancer cells found in other parts of the body
  • A suitable donor must be available, which can be a family member or unrelated person whose tissue type matches the patient's tissue type closely enough
  • The donor cells can come from bone marrow, which is the spongy tissue inside bones, from peripheral blood, which is blood taken from a vein, or from cord blood, which is blood collected from the umbilical cord after birth
  • Female patients who are able to become pregnant must have a negative pregnancy test before joining the study
  • All patients must agree to use effective birth control methods during the study
  • The patient or their parent or legal guardian must sign a consent form agreeing to participate in the study
  • The patient must not have had any other types of cancer in the past

Who cannot join the trial?

1 criterion

  • No exclusion criteria have been specified in the available study information. Please consult with the study team or your doctor to understand if there are any specific conditions or situations that would prevent participation in this clinical trial.
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Investigated drugs

Ruxolitinib is a medicine used in combination with corticosteroids to treat a complication that can occur after stem cell transplant called graft-versus-host disease. This complication happens when the transplanted cells attack the patient's body.

Blinatumomab is a medicine used after stem cell transplant to help prevent the leukemia from coming back. It works by helping the body's immune system find and destroy cancer cells that have a protein called CD19 on their surface.

Etoposide is a chemotherapy medicine used as part of the preparation treatment before stem cell transplant. It helps destroy cancer cells and makes room in the bone marrow for the new stem cells to grow.

Corticosteroids are medicines used to treat graft-versus-host disease, a complication that can occur after stem cell transplant. They work by reducing inflammation and calming down the immune system.

Total body irradiation is a treatment that uses radiation to destroy cancer cells throughout the entire body. It is given before stem cell transplant to prepare the body to receive the new stem cells.

What is already known about the treatment

  • Ruxolitinib

    Ruxolitinib is a medication given by mouth in tablet form that is used to treat acute graft-versus-host disease, a serious complication that can occur after stem cell transplantation where the donated immune cells attack the patient's body. This drug works by blocking specific proteins called JAK1 and JAK2, which are involved in the inflammatory response that causes tissue damage in this condition. It belongs to a class of medicines called JAK inhibitors and is being studied in combination with corticosteroids to improve treatment outcomes in patients who develop this complication after receiving a stem cell transplant.

  • Blinatumomab

    Blinatumomab is a medication administered through continuous intravenous infusion that is used to treat certain types of acute lymphoblastic leukemia, specifically those with CD19-positive B-cells. This drug is a special type of antibody that works by connecting cancer cells to the patient's own immune cells, helping the immune system recognize and destroy the leukemia cells more effectively. It belongs to a class of medicines called bispecific T-cell engager antibodies and is being studied as a maintenance therapy after stem cell transplantation in young children to help prevent the cancer from returning.

Investigated diseases

  • Acute Lymphoblastic Leukemia

    Acute lymphoblastic leukemia is a type of cancer that affects the blood and bone marrow. It occurs when the bone marrow produces too many immature white blood cells called lymphoblasts. These abnormal cells crowd out healthy blood cells and spread through the bloodstream. The disease progresses rapidly if left untreated. It primarily affects children but can also occur in adults. The condition interferes with the body's ability to fight infections and produce normal blood cells.

  • Acute Graft-Versus-Host Disease

    Acute graft-versus-host disease is a complication that can occur after a stem cell or bone marrow transplant. It happens when the donated immune cells recognize the recipient's body tissues as foreign and attack them. The condition typically develops within the first few months after transplantation. It commonly affects the skin, liver, and digestive system. The severity can range from mild to severe, impacting multiple organs. The disease results from an immune reaction between the transplanted cells and the host body.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2025-522052-13-00Protocol codeFORUM2Estimated enrolment1 213 patientsSponsorOspedale Pediatrico Bambino Gesu

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