Who can enter
- Children and adolescents who have recently been told they have chronic graft-versus-host disease (cGVHD)
- Age: 12 years and older
Goal
With this study, we want to show that taking the drug belumosudil together with prednisone may work better than taking prednisone alone. This means that participants taking both drugs together may have a greater chance of living longer without serious problems, compared to participants taking prednisone alone.
Background
Chronic graft-versus-host disease (cGVHD) is a complex condition that can occur after a stem cell or bone marrow transplant. In this condition, the transplanted cells from the donor (the “graft”) do not recognize the body of the recipient (the “host”) as their own and attack it. This is caused by an immune reaction.
In cGVHD, a large number of organs and systems in the body can be affected. The disease can cause different symptoms depending on which organs are involved. It can affect the skin, mouth, eyes, digestive system, liver and lungs, among others.
The condition is characterized by a combination of immune responses and fibrosis, meaning excessive scar tissue, which can interfere with the normal functioning of organs.
In treating cGVHD, doctors and scientists have already tried using different drugs together in the hope that these combinations would work better than standard treatment. belumosudil has been shown to be effective in patients with cGVHD, especially those who saw no improvement with the usual treatments. Combining belumosudil with some type of anti-inflammatory drug, such as prednisone, may yield better results.
There are two groups in the study, one receiving the treatment and one receiving the placebo.
Belumosudil is a new drug for participants who have just been told they have cGVHD. It helps reduce inflammation in the body and blocks certain signals that can make the disease worse. In the study, participants are randomly, determined by a computer, given either belumosudil with prednisone or a placebo with prednisone. They take this every day until the end of the study.
This drug is already approved in several countries for adults and children as young as 12 years old who have already had at least two other treatments. Previous scientific studies found that most participants tolerated belumosudil well.
There are some risks. Belumosudil can affect the immune system, which may increase the risk of infections and other conditions. Some participants saw a temporary increase in liver enzymes, but this usually improved with proper treatment. It is also important for women not to become pregnant during treatment with belumosudil.
In this study, we are comparing belumosudil with a placebo to see how well it works. A placebo helps us understand what the effect of the real drug is compared to just treatment with prednisone.