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Clinical study of belumosudil in children with chronic graft-versus-host disease.
Recruiting
Who can enter
Children aged 1 to less than 18 years with chronic graft-versus-host disease (cGVHD)
Who have previously received at least 2 systemic treatments for cGVHD
Goal
The goal of this study is to determine if belumosudil is safe and effective in children with cGVHD, and to find the right dose for children under 12 years of age.
Background
Chronic graft-versus-host disease (cGVHD) is an immune-mediated and fibrotic condition that can occur after stem cell or bone marrow transplantation, where the transplanted donor cells attack multiple organs in the recipient's body.
Currently, corticosteroids are the standard treatment for newly diagnosed cGVHD, but there is only one alternative for patients under 12 years who experience relapse or worsening of their condition. The drug belumosudil is already approved in several countries for treating cGVHD in patients 12 years or older who have failed other treatments. This study will help us understand if it can also help younger children.
Belumosudil works by reducing inflammation in the body and blocking abnormal pro-fibrotic signals that lead to tissue scarring. The medication will be given once daily, with younger children receiving a liquid form and children 12 years or older having the choice between liquid or tablet form.
In order to participate in a study please refer to your/your child’s doctor.
Last reviewed
January 30, 2026
Study details
- Study details
Official title
An open-label, multicenter, phase 1/2 study of belumosudil in pediatric subjects aged 1 to <18 years requiring systemic therapy for active moderate to severe chronic graft[1]versus-host disease (cGVHD)
Disease
Chronic graft-versus-host disease (cGVHD), a complication that can occur after stem cell or bone marrow transplantation
Phase
1/2
Maximum number of patients
25 to 37 children, of whom participants will be enrolled across 11 to 13 countries, of whom approximately 3 participants are expected to participate in the Netherlands
Start date
January 30, 2026
Status
Open for inclusion
Local principal investigator
Dr. Lindemans
Sponsor
Sanofi
Approval
The study of this new treatment has been reviewed by an accredited medical research ethics committee. This committee has decided that it is justified to ask patients to participate in this study. More information can be found at: CCMO.
Trial registry number
EU Clinical Trials: 2024-511508-18
The above information is intended as a brief summary only and may not reflect the most up-to-date information. For full details and the current status of a protocol, physicians can contact the Princess Máxima Center directly.