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Study on the Effectiveness of Mesenchymal Stromal Cells in Children with Steroid-Refractory Acute Graft-Versus-Host Disease

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 known as steroid-refractory acute graft-versus-host disease (SR aGvHD), which can occur after a patient receives a stem cell transplant from a donor. This condition happens when the donor's immune cells attack the recipient's body. The trial will compare the effectiveness and safety of a new treatment using mesenchymal stromal cells, also known by the code name MC0518, against the best available therapies currently used for this condition. Mesenchymal stromal cells are a type of cell therapy derived from bone marrow that is cultured outside the body before being infused into the patient.

Participants in the study will receive either the new treatment or one of the existing therapies, which may include medications like infliximab, anti-T lymphocyte immunoglobulin (from rabbits), methoxsalen, ruxolitinib, or etanercept. These treatments are administered in different ways, such as through intravenous infusion or oral intake, depending on the specific medication. The purpose of the study is to evaluate how well these treatments work in improving the condition by assessing the overall response rate at specific intervals, such as 28 days after starting the treatment.

The trial will follow participants over a period of time to monitor their response to the treatment, any side effects, and overall health outcomes. The study aims to provide valuable information on the best treatment options for children and adolescents with SR aGvHD, potentially improving their quality of life and long-term health. Participants will be closely monitored by healthcare professionals throughout the study to ensure their safety and well-being.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and randomization

    Upon joining the trial, the participant is randomly assigned to receive either mesenchymal stromal cells (MC0518) or the best available therapy (BAT) for treating steroid-refractory acute graft-versus-host disease (aGvHD).

  2. Step 2

    Initial treatment phase

    The participant begins treatment with the assigned therapy. If receiving MC0518, the treatment involves intravenous administration of mesenchymal stromal cells.

    If assigned to BAT, the treatment may include one or more of the following medications: infliximab (intravenous), antithymocyte immunoglobulin (rabbit) (intravenous), methoxsalen (extracorporeal), ruxolitinib (oral), or etanercept (subcutaneous injection).

  3. Step 3

    Monitoring and assessment

    The participant's response to treatment is assessed at various intervals, including Visit Day 8, Visit Day 15, Visit Day 22, and Visit Day 28. The primary focus is on the overall response, which includes complete or partial resolution of aGvHD symptoms.

    Additional assessments occur at Visit Day 60, Visit Day 100, and Visit Day 180 to evaluate the participant's condition and any changes in aGvHD symptoms.

  4. Step 4

    Follow-up and long-term evaluation

    The participant continues to be monitored for up to 24 months to assess long-term outcomes, including overall survival, incidence of chronic graft-versus-host disease (cGvHD), and any adverse events.

    Quality of life is evaluated using the Paediatric Quality of Life Inventory at specified intervals to understand the impact of the treatment on daily living.

Who can join the trial?

8 criteria

  • The participant must have had a previous **allogeneic HSCT**. This means they received a stem cell transplant from a donor to treat certain diseases.
  • The participant must have been diagnosed with **Grade II to IV aGvHD**. This is a condition where the transplanted cells attack the body, and it is classified into grades based on severity.
  • The participant must have experienced **failure of previous first-line aGvHD treatment**. This means the initial treatment did not work, and the condition either got worse or did not improve as expected.
  • The participant must be between **28 days and 18 years old** and weigh at least **3.2 kg** at the time of screening.
  • The participant must have an estimated life expectancy of more than **28 days**.
  • If the participant is a female who can have children, she must agree to use a **highly effective contraceptive method** during the trial.
  • If the participant is a fertile male, he must agree to either abstain from sexual activity or use a **condom** during sexual activity. If his partner can have children, she must also use an additional highly effective contraceptive method.
  • A written **informed consent** must be obtained from the participant's parent(s) or legal guardian(s). If applicable, the participant must also give their assent, which means they agree to participate.

Who cannot join the trial?

3 criteria

  • Patients who have not been diagnosed with **steroid-refractory acute graft-versus-host disease**. This is a condition where the body reacts against a transplant, and it does not improve with steroid treatment.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the vulnerable population selected for the study.
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Investigated drugs

  • MC0518

    is a type of therapy that uses mesenchymal stromal cells. These cells are special because they can help reduce inflammation and support healing in the body. In this trial, MC0518 is being tested to see if it can help children who have a condition called steroid-refractory acute graft-versus-host disease (SR aGvHD). This condition can happen after a stem cell transplant and is difficult to treat with standard medications.

  • Best Available Therapy (BAT)

    refers to the most effective treatment options currently available for managing steroid-refractory acute graft-versus-host disease. This can include a variety of medications and therapies that doctors use based on their experience and the latest research to help control the symptoms of the disease. The trial is comparing the effectiveness of MC0518 against these existing treatments to see which works better for children with this condition.

What is already known about the treatment

Mesenchymal Stromal Cells MC0518 – This medication is administered through intravenous infusion and is currently being studied in clinical trials for its effectiveness in treating steroid-refractory acute graft-versus-host disease in children. It is not yet widely used in standard medical practice, as it is still under investigation. The main therapeutic indication is for patients who do not respond to standard steroid treatments after receiving an allogeneic stem cell transplant. At the molecular level, mesenchymal stromal cells are believed to modulate the immune response, reducing inflammation and promoting tissue repair. They are classified as a type of cell therapy, which involves using living cells to treat or prevent disease.

Investigated diseases

Steroid-Refractory Acute Graft-Versus-Host Disease – This condition occurs when the immune cells from a donor attack the recipient's body after a stem cell or bone marrow transplant, and it does not respond to standard steroid treatments. It primarily affects the skin, liver, and gastrointestinal tract, leading to symptoms such as rash, jaundice, and diarrhea. The disease progresses as the immune response continues to damage these organs, potentially leading to severe complications. The severity of symptoms can vary, and the condition is classified into different grades based on the extent of organ involvement. Management focuses on controlling the immune response to prevent further damage.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2023-503952-28-00Protocol codeMC MSC.2/aGvHDEstimated enrolment54 patientsSponsorMedac Gesellschaft fuer klinische Spezialprapaerate mbH

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