A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK)
This study, called schoolROCK, is testing a new medicine, Belumosudil, in children aged 1 to 18 who have chronic graft versus host disease (cGVHD). This is a condition that can happen after a stem cell transplant. We're looking for children whose cGVHD hasn't improved with at least two other types of treatment. The study has two parts: the first part is for younger children (1 to under 12) to figure out the best dose. The second part will then check how safe and effective the medicine is for all participants up to 18 years old. We hope this medicine can offer a new option for these children.
At a glance
What is this study about?
This study, called schoolROCK, is for children who have a condition called chronic graft versus host disease (cGVHD). This can happen after a stem cell transplant, where the new immune cells from the donor start to attack the patient's body. Despite being a treatment for serious illnesses, cGVHD can be quite challenging for children and their families, impacting বিভিন্ন parts of their body.
We are looking at a new medicine called Belumosudil. It's hoped that this medicine can help manage cGVHD symptoms in children who haven't responded well to at least two other previous treatments. The study aims to find out the right dose for children and then to see how safe and effective it is. By taking part, children might get access to a new treatment option that could potentially improve their condition.
It's important to understand that this is a research study, and while we hope Belumosudil will be helpful, we are still learning about it. The study will carefully monitor participants to understand how the medicine works in children and to ensure their safety and well-being throughout the process. This kind of research is vital for finding new and better ways to treat serious conditions like cGVHD.
Key takeaways
- This study is for children aged 1 to under 18 with chronic graft versus host disease (cGVHD).
- It tests a new medicine called Belumosudil for cGVHD that hasn't responded to at least two other treatments.
- The study aims to find the right dose for children and check its safety and effectiveness.
- Participation involves screening, taking the medicine, and regular follow-up visits.
- Your child could receive a new treatment option, but potential risks will be carefully monitored.
- You can withdraw your child from the study at any time without affecting their regular care.
Who may be eligible?
To be considered for this study, your child needs to be between 1 and under 18 years old. They must have undergone a stem cell transplant and currently have moderate to severe chronic graft versus host disease (cGVHD) that has not improved with at least two previous treatments. There's a limit of no more than five previous treatments for cGVHD.
Your child should be generally stable otherwise, meaning no rapidly worsening underlying disease, active untreated infections, or other serious health issues that the study doctors believe would make their participation unsafe. They'll also need to have a good performance score, which means they are able to carry out most daily activities.
Some things would prevent your child from joining. For example, if they have other types of cancer diagnosed in the last three years (other than the one they had the transplant for), or if they have certain severe lung problems. The study doctor will check all these details carefully to see if your child is a good fit.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child aged between 1 and under 18 years old?
- Has your child had a stem cell transplant?
- Does your child have moderate to severe cGVHD that hasn't improved with at least two other treatments?
- Has your child received no more than five previous treatments for cGVHD?
- Is your child generally well enough to participate in daily activities?
What does participation involve?
If your child takes part, they'll first go through a screening period lasting up to 4 weeks. During this time, doctors will do checks and tests to make sure the study is right for them. If they join, your child will then start taking the study medicine, Belumosudil. This medicine can be taken by mouth or through a feeding tube.
Treatment will continue for as long as it's helping and safe for your child. This means it could stop if their cGVHD gets worse, if the original disease returns, if they need a new cGVHD treatment, or if they experience serious side effects. Either you or the doctor can decide to stop treatment at any time. Once treatment stops, there will be a 30-day period where their safety is closely monitored.
After this, there will be long-term follow-up visits until the study officially ends or if your child passes away. The whole study could last for up to 3 years after the last person joins. During the study, there will be regular hospital or clinic visits for check-ups and to see how your child is doing and if the medicine is working.
Potential risks and benefits
Locations (30)
- Children's Hospital Los Angeles- Site Number : 8400009Verified postcodeLos Angeles, United States· Recruiting
- Children's National Medical Center - Washington- Site Number : 8400005Verified postcodeWashington D.C., United States· Recruiting
- Memorial Sloan Kettering Cancer Center - New York - York Avenue- Site Number : 8400001Verified postcodeNew York, United States· Recruiting
- Texas Children's Hospital- Site Number : 8400008Verified postcodeHouston, United States· Recruiting
- Investigational Site Number : 0560003Verified postcodeGhent, Belgium· Recruiting
- Investigational Site Number : 0560001Verified postcodeLeuven, Belgium· Recruiting
- Investigational Site Number : 1240001Verified postcodeVancouver, Canada· Recruiting
- Investigational Site Number : 1240002Verified postcodeToronto, Canada· Recruiting
- Investigational Site Number : 1560003Verified postcodeBeijing, China· Recruiting
- Investigational Site Number : 1560001Verified postcodeShanghai, China· Recruiting
- Investigational Site Number : 1560004Verified postcodeShenzhen, China· Recruiting
- Investigational Site Number : 1560002Verified postcodeSuzhou, China· Recruiting
Common questions
What is chronic graft versus host disease (cGVHD)?
cGVHD is a condition that can happen after a stem cell transplant. It’s when the donor’s immune cells see the patient’s body as different and start to attack it, causing different problems in the body.
What is Belumosudil?
Belumosudil is the name of the new medicine being tested in this study. It's hoped to help control the immune system and reduce cGVHD symptoms. It’s an investigational medicine, meaning it’s not yet widely available.
Will my child definitely get the new medicine?
Yes, everyone in this study will receive Belumosudil. There isn't a placebo (dummy treatment) group in this particular study.
How long will my child be in the study?
Your child will receive treatment for as long as it's safe and helping their condition. There will be 30 days of safety follow-up after treatment stops, and then long-term follow-up until the study ends. The maximum total study time could be several years.
What if the medicine doesn't work or causes problems?
The study team will closely monitor your child. If their cGVHD gets worse, if they have serious side effects, or for any other reason, the treatment can be stopped. You also have the right to withdraw your child from the study at any time.
How to find out more
Trial Transparency email recommended (Toll free for US & Canada)
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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