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Study of belumosudil in children aged 1 to 18 years with moderate to severe chronic graft versus host disease requiring systemic treatment

Verified siteInvestigationalNo 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 children with chronic graft versus host disease (cGVHD), a condition that can occur after receiving cells from a donor during a stem cell transplant. The disease happens when the donated cells attack the recipient's body, causing inflammation and damage to various organs. The study will test a medication called belumosudil, which is available as both film-coated tablets and oral suspension that can be taken by mouth or through a feeding tube.

The study aims to find the right dose of belumosudil for children and determine how well it works in treating moderate to severe chronic graft versus host disease. The research is divided into two parts. In the first phase, researchers will work to establish the appropriate dose for children. In the second phase, they will evaluate how many children respond positively to the treatment.

During the study, participants will receive belumosudil and will be monitored regularly to check their response to the treatment. The medication can be taken either as tablets or as a liquid suspension, depending on what works best for each child. The study will track how well the medication helps to control the symptoms of chronic graft versus host disease and will also monitor the safety of the treatment in children.

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

    Initial evaluation and medication start

    You will begin taking belumosudil, a medication for treating chronic graft versus host disease (cGVHD).

    The medication can be taken as a film-coated tablet or oral suspension.

    The medicine can be taken orally, through a nasogastric tube, or through a feeding tube (PEG).

    Your current medications for cGVHD must remain stable for at least 2 weeks before starting this treatment.

  2. Step 2

    Treatment period - Phase 1

    If you are between 1 and 12 years old, you may participate in Phase 1 of the study.

    During this phase, doctors will determine the most appropriate dose of the medication for children.

    Your health status will be monitored for any side effects or reactions to the medication.

    The effectiveness of the treatment will be evaluated based on your response to the medication.

  3. Step 3

    Treatment period - Phase 2

    If you are between 1 and 18 years old, you may participate in Phase 2 of the study.

    The treatment response will be evaluated by Week 25 or Cycle 7 Day 1, whichever comes first.

    Doctors will monitor how your body responds to the medication in different affected areas.

    Your overall health status and survival will be tracked throughout the study period.

  4. Step 4

    Ongoing monitoring

    Regular check-ups will assess your response to the treatment.

    Any side effects or health changes will be documented.

    The treatment will continue as long as it provides benefit and is safe.

    The study is expected to continue until February 2028.

Who can join the trial?

11 criteria

  • Age requirement: For Phase 1 - between 1 and 12 years old; For Phase 2 - between 1 and 18 years old
  • Must have a life expectancy of more than 6 months
  • Must be able to take medication either by mouth or through a feeding tube (nasogastric tube)
  • Must have received a stem cell transplant from a donor (allogeneic HCT)
  • Must have active moderate to severe chronic graft versus host disease that requires systemic treatment (medication that affects the entire body)
  • Current treatment must not be working or disease has come back after at least 2 different treatments
  • Must have received between 2 and 5 different previous treatments
  • If taking steroids, the dose must remain unchanged for at least 2 weeks before starting the study medication
  • Must have a performance score of 60 or higher on the Lansky-Play scale (for ages 16 and under) or Karnofsky scale (for ages over 16) - these scales measure how well a person can perform daily activities
  • Must weigh at least 8 kilograms
  • Sexually active participants must use appropriate contraception methods according to local guidelines for clinical trials

Who cannot join the trial?

14 criteria

  • Active and uncontrolled bacterial, fungal, or viral infections that require treatment
  • Severe liver problems, including abnormal liver function tests that are more than 3 times the normal limit
  • Severe kidney problems requiring dialysis (a procedure to filter blood when kidneys don't work properly)
  • Uncontrolled heart conditions including heart failure, irregular heartbeat, or recent heart attack (within past 6 months)
  • Active or untreated cancer other than the condition being studied
  • Pregnancy or breastfeeding
  • Use of other experimental medications within 30 days before starting this study
  • History of organ transplant rejection
  • Severe mental health conditions that could interfere with following study procedures
  • Known allergic reactions to similar medications
  • Unable to swallow oral medications
  • Participation in another clinical trial at the same time
  • Blood disorders that result in very low blood cell counts
  • History of serious bleeding problems
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Investigated drugs

Belumosudil is a medication being studied for treating chronic graft versus host disease (cGVHD) in children. This medication helps manage the immune system's response when it attacks healthy tissues after a stem cell or bone marrow transplant. It works by blocking specific proteins that contribute to inflammation and tissue damage in cGVHD, potentially helping to reduce symptoms like skin problems, mouth sores, and other complications that can occur after transplantation.

What is already known about the treatment

Belumosudil – An oral medication administered in tablet form that represents a novel treatment approach for chronic graft versus host disease (cGVHD). This drug belongs to the class of kinase inhibitors, specifically targeting ROCK2 (Rho-associated coiled-coil containing protein kinase 2), which plays a crucial role in immune system regulation. Belumosudil works by modulating inflammatory responses and reducing the severity of immune reactions that occur when transplanted donor cells attack the recipient's tissues. It is currently being investigated in clinical trials for pediatric patients with moderate to severe chronic graft versus host disease who require systemic treatment, showing promise as a targeted therapy option for this challenging condition.

Investigated diseases

Chronic Graft-versus-Host Disease - A condition that occurs after bone marrow or stem cell transplantation where the donated immune cells attack the recipient's body tissues. The disease typically develops within the first year after transplantation and can affect multiple organs including the skin, mouth, eyes, liver, lungs, and joints. The condition causes inflammation and fibrosis in affected tissues, leading to various symptoms such as skin changes, dry mouth, vision problems, and joint stiffness. This disease can range from mild to severe and may develop gradually over time. The immune system's response creates ongoing inflammation that can cause tissue damage and scarring in affected areas.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-511508-18-00Protocol codeDFI17893Estimated enrolment50 patientsSponsorSanofi-Aventis Recherche & Developpement

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