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Not yet recruitingRare disease

Study of belumosudil versus drug combination in patients aged 12 years and older with chronic graft‑versus‑host disease refractory after prior therapy

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

The study focuses on chronic graft-versus-host disease, a rare condition that can occur after a stem‑cell transplant when the donor’s immune cells attack the recipient’s body. Participants must be at least 12 years old and have disease that has not improved after two to five previous treatments. The purpose of the study is to compare the effectiveness and safety of the oral drug belumosudil with the best available therapy, which may include medicines such as sirolimus, ibrutinib, everolimus, imatinib mesilate, rituximab, carfilzomib, pentostatin, methotrexate sodium, bortezomib, ixazomib citrate, and mycophenolate mofetil.

Participants are randomly assigned to receive either belumosudil tablets taken by mouth or one of the comparator medicines chosen by their doctor. The study lasts about 24 weeks, during which regular clinic visits are scheduled to check symptoms, perform blood tests, and assess overall health. Researchers look at the overall response rate, which means the percentage of people whose disease shows improvement, using standard doctor‑approved guidelines (NIH consensus response criteria). Systemic therapy refers to medicines that work throughout the whole body, and any need for new systemic treatment, relapse of the underlying disease, or death is recorded.

Throughout the trial, safety is closely monitored; blood samples are taken to measure drug levels and to watch for side effects. Participants can stop the study at any time if they experience problems. The information gathered will help determine whether belumosudil works better and is safe for people with this condition.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Baseline assessment and consent

    After joining the study you will complete a consent form and undergo baseline assessments, which may include physical examination, blood tests, and questionnaires about your symptoms.

    The information collected will be used to determine eligibility and to serve as a reference point for future evaluations.

  2. Step 2

    Randomization

    Once baseline data are confirmed you will be randomly assigned to one of two groups: the belumosudil group or the best available therapy (bat) group.

    Randomization ensures that the assignment is unbiased and that the groups are comparable.

  3. Step 3

    Start of study medication

    If you are assigned to the belumosudil group you will begin taking belumosudil tablets that contain 400 mg of the active ingredient. the tablets are taken by mouth; the exact frequency and duration will be specified in the study instructions, typically daily for up to 24 weeks.

    If you are assigned to the bat group you will receive one of the comparator medicines that are commonly used for chronic graft‑versus‑host disease. these may include oral drugs such as sirolimus, ibrutinib, everolimus, imatinib mesilate, methotrexate sodium, mycophenolate mofetil, pentostatin, or ixazomib, and infusion drugs such as rituximab, carfilzomib, bortezomib. each drug will be given by the route indicated (oral use or infusion) and at the dose normally used for this condition, as directed by the study physician.

  4. Step 4

    Regular clinic visits

    You will attend scheduled clinic visits to monitor safety and efficacy. typical visits occur at week 4, week 8, week 12, and week 24 after starting the medication.

    During each visit blood samples will be drawn, vital signs checked, and symptom questionnaires completed.

  5. Step 5

    Response and safety monitoring

    The study team will evaluate your response using the modified lee cgvhd symptom scale and other criteria. a response is considered a reduction of at least six points on the scale or an overall improvement classified as complete response (cr) or partial response (pr).

    Any adverse events, including serious adverse events, will be recorded and managed according to the study protocol.

  6. Step 6

    Continuation criteria

    You will continue the assigned medication until week 24, unless one of the following occurs first: the need to start a new systemic treatment for cgvhd, disease relapse or recurrence, or death.

    If a new systemic therapy becomes necessary, the study medication will be stopped and the date of the change will be recorded.

  7. Step 7

    Final evaluation at week 24

    At week 24 a comprehensive assessment will be performed to determine the overall response rate, changes in symptom scores, and any reductions in concomitant steroid or calcineurin inhibitor doses.

    Blood samples will also be taken to measure belumosudil plasma concentrations (for participants receiving belumosudil).

  8. Step 8

    Long‑term follow‑up

    After the week‑24 assessment you may be asked to continue providing safety information for a period defined by the study, which may extend until the overall study end date.

    This follow‑up helps capture any late‑onset adverse events or disease outcomes.

Who can join the trial?

10 criteria

  • Participant must be at least 12 years old when signing the informed consent.
  • Both men and women must use birth control methods that follow local rules for clinical studies.
  • Participant must have had an allo‑HCT – a stem cell transplant using cells from a donor.
  • Participant must have active moderate to severe chronic graft‑versus‑host disease (cGVHD) at the time of enrollment, defined by the NIH (National Institutes of Health) diagnosis and staging guidelines, and the doctor must think a new systemic therapy (medicine that works throughout the whole body) is needed.
  • Participant must be on a stable dose of a calcineurin inhibitor (CNI) and/or a corticosteroid (CS) such as prednisone (less than 1 mg per kilogram of body weight per day) for at least two weeks before randomization.
  • The cGVHD must be refractory (does not respond) or have come back after at least two previous lines of systemic treatment; the total number of prior systemic therapies must be between 2 and 5.
  • Participant must have already received ruxolitinib for cGVHD unless the investigator decides this drug is not suitable for them.
  • Participant and/or their legally authorized representative must agree to receive one of the recommended best available therapy (BAT) options, such as ECP, low‑dose methotrexate (MTX), mycophenolate mofetil (MMF), rituximab, mTOR inhibitors (e.g., sirolimus or everolimus), imatinib, ibrutinib, proteasome inhibitors, or pentostatin.
  • Participant must weigh at least 30 kg.
  • Participant must have a life expectancy (expected time to live) of more than six months.

Who cannot join the trial?

18 criteria

  • Any sign that the original disease has gotten worse again or come back, or any evidence of a condition called post‑transplant lymphoproliferative disease after the most recent allo‑HCT (allogeneic stem‑cell transplant).
  • A lung function test called FEV1 that is 39% or lower of the normal value, or a lung rating of 3 based on the 2014 NIH guidelines, indicating very poor lung health.
  • Lab results showing any of the following:
    • Very low white blood cells (absolute neutrophil count) – less than 1.5 × 10⁹/L for people under 18 years old or less than 1.0 × 10⁹/L for adults.
    • Very low platelets (platelet count) – less than 50 × 10⁹/L for people under 18 or less than 25 × 10⁹/L for adults.
    • High liver enzymes (ALT and/or AST) more than three times the normal upper limit (or more than five times if the rise is caused by the disease being studied).
    • High bilirubin (total bilirubin) more than 1.5 times the normal upper limit (or more than three times if related to the disease).
    • Poor kidney function (eGFR) less than 30 mL/min/1.73 m².
    • Severe liver disease classified as Child‑Pugh C or worse, unless it is a temporary liver problem caused by the disease being studied.
    • Having an active viral infection at the time of screening.
    • Being diagnosed with or treated for another cancer within the past 3 years, except for completely removed basal or squamous cell skin cancers, non‑invasive cancers, or low‑risk prostate cancer that has been cured.
    • Any of the following recent systemic treatments for the disease being studied:
      • Starting a new systemic therapy within 14 days before randomization.
      • Taking the drug ruxolitinib without being able to taper and stop it within 14 days after the first dose of the study medication, and without increasing the dose during the 14‑day period before randomization.
      • Taking other systemic therapies (including experimental drugs) without a washout period of at least 14 days (or 5 half‑lives, whichever is shorter) before the first study dose.
      • Having taken the study drug belumosudil before.
      • Scoring less than 60 on the Karnofsky Performance Scale (for people 16 years or older) or the Lansky Performance Score (for children under 16), which means a low ability to carry out daily activities.
      • Having an uncontrolled chronic infection that requires antibiotics, antivirals, or antifungal medicines within 14 days before randomization.
      • Having gastrointestinal (GI) problems unrelated to the disease that could affect how the study drug is absorbed, such as ulcerative disease, malabsorption syndrome, uncontrolled nausea, vomiting, diarrhea, or removal of part of the small intestine.
      • Receiving a live or live‑attenuated vaccine within 28 days (or five vaccine half‑lives, whichever is longer) before starting the study treatment, and continuing such vaccines until the study medication is stopped.
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Investigated drugs

  • Belumosudil

    is an oral tablet taken by mouth. It works by blocking a protein that helps control the immune system, which can reduce the harmful immune activity that causes chronic graft‑versus‑host disease (cGVHD). In this study it is the new treatment being tested to see if it works better than the other medicines that patients may already be using.

  • Sirolimus

    is a pill that suppresses the immune system by stopping certain immune cells from growing. Doctors often use it to prevent organ rejection after a transplant. In the trial it is one of the existing medicines that could be chosen as part of the best available therapy for cGVHD.

  • Ibrutinib

    is an oral drug that blocks a signaling pathway important for certain immune cells and cancer cells. It is approved for some blood cancers and for chronic graft‑versus‑host disease. In this study it may be used as a comparator option for patients who need additional therapy.

  • Everolimus

    is a tablet that reduces activity of the immune system by inhibiting a key growth‑regulating protein. It is used to prevent organ rejection and to treat some cancers. Here it can be selected as part of the standard treatments against cGVHD.

  • Imatinib

    is a pill that blocks a specific enzyme involved in the growth of certain abnormal cells. It is best known for treating chronic myeloid leukemia but is also used for some forms of cGVHD. In the trial it may be one of the medicines offered as best available therapy.

  • Rituximab

    is given by infusion into a vein. It is an antibody that targets a protein on B‑cells, a type of immune cell, leading to their removal. It is used for various autoimmune diseases and some cancers. In this study it can be chosen as a treatment option for cGVHD.

What is already known about the treatment

  • Sirolimus

    This drug is taken by mouth as a tablet and is an approved medication used mainly to prevent organ transplant rejection. It works by blocking a protein called mTOR, which helps stop immune cells from becoming over‑active. Sirolimus belongs to the class of mTOR inhibitors and is listed in medical literature as a standard immunosuppressive agent.

  • Ibrutinib

    Ibrutinib is an oral tablet that has been approved for several blood cancers such as chronic lymphocytic leukemia. It blocks an enzyme called Bruton's tyrosine kinase, which slows the growth of malignant B‑cells. The drug is classified as a BTK (Bruton’s tyrosine kinase) inhibitor and is widely referenced in cancer treatment guidelines.

  • Everolimus

    Everolimus is taken by mouth and is approved for preventing organ transplant rejection and for treating certain cancers. It also blocks the mTOR pathway, reducing the activity of immune and tumor cells. This medication is part of the mTOR inhibitor class and is well‑documented in current medical practice.

  • Imatinib

    Imatinib mesylate comes as oral tablets and is approved for chronic myeloid leukemia and some gastrointestinal tumors. It stops a protein called BCR‑ABL tyrosine kinase, which stops cancer cells from multiplying. Imatinib is a tyrosine‑kinase inhibitor and has orphan‑drug designation for its use in rare diseases.

  • Rituximab

    Rituximab is given as an infusion solution and is an approved monoclonal antibody used for certain lymphomas and autoimmune disorders. It binds to a marker called CD20 on B‑cells, leading to their removal from the bloodstream. The drug belongs to the anti‑CD20 monoclonal antibody class and holds orphan‑drug status for some rare conditions.

  • Carfilzomib

    Carfilzomib is administered by intravenous infusion and is approved for treating multiple myeloma. It works by permanently blocking the proteasome, a cell structure that breaks down proteins, causing cancer cells to die. This medication is classified as a proteasome inhibitor and is widely used in hematologic oncology.

Investigated diseases

Chronic graft‑versus‑host disease - Chronic graft‑versus‑host disease is an immune‑mediated condition that can develop after an allogeneic stem cell transplant, when donor immune cells recognize the recipient’s tissues as foreign. It typically appears three months or more after transplantation and may involve the skin, liver, eyes, mouth, lungs, gastrointestinal tract, and other organs. The disease often starts with mild symptoms such as rash or dry eyes, and over time can become more extensive, affecting multiple organ systems. In some people, the inflammation and fibrosis progress gradually, leading to functional impairment of the involved organs. The severity can fluctuate, with periods of worsening followed by partial improvement.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2026-525913-30-00Protocol codeEFC22965Estimated enrolment464 patientsSponsorSanofi-Aventis Recherche & Developpement

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