Phase 1/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
This study is a clinical trial focusing on a medication called ibrutinib (also known as IMBRUVICA) for children and young people suffering from chronic Graft Versus Host Disease (cGVHD). cGVHD is a serious complication that can happen after a stem cell transplant. The trial has two main parts. Part A will help doctors find the best dose of ibrutinib for children aged 1 to 12 years. Part B will then look at both the safety and how well ibrutinib works in a wider age range of children and young people, from 1 to 22 years old. Researchers will monitor for any side effects and see if the treatment helps improve their cGVHD symptoms. They will also look at how the medicine affects their growth and development.
At a glance
What is this study about?
This study is a research project designed to help children and young people who have a condition called chronic Graft Versus Host Disease (cGVHD). cGVHD is a serious problem that can sometimes happen after someone has had a stem cell transplant, often to treat cancers like leukaemia. It occurs when the donated healthy cells start to see the patient's own body as foreign and attack it.
The trial is testing a medicine called ibrutinib, which is already used for some other conditions in adults. The main goals of this study are to figure out the right amount (dose) of ibrutinib to give to children and young people, to make sure it's safe, and to see how well it works in treating their cGVHD. The study will look carefully at whether the medicine helps reduce the symptoms of cGVHD and improves their overall health.
Researchers will be splitting the study into two parts. In Part A, they'll focus on finding the best dose for younger children (aged 1 to under 12 years old). In Part B, they'll then study both the safety and effectiveness of this medicine in a broader group of children and young people (aged 1 to under 22 years old). This includes checking for any side effects, observing how the medicine affects their body, and monitoring their growth and development over time. They will also see how many patients respond to the treatment and how long that response lasts.
Key takeaways
- The study is for children and young people (aged 1 to under 22) with chronic Graft Versus Host Disease (cGVHD).
- It aims to find the right dose of ibrutinib and check its safety and effectiveness.
- Children will take ibrutinib tablets and possibly other medicines like prednisone.
- Regular health checks, blood tests, and monitoring of growth will be part of the study.
- Researchers will observe how well the treatment helps cGVHD symptoms and for how long.
Who may be eligible?
This study is looking for children and young people who have chronic Graft Versus Host Disease (cGVHD). While the original information suggests a broad age range for eligibility, the details of the study clearly state it's for those aged 1 year up to 21 years old (so, less than 22 years).
If your child has cGVHD and falls within this age group, they might be able to take part. Both boys and girls are welcome to join the study.
To find out if your child definitely qualifies, a doctor will need to check their full medical history and current health status against specific criteria. This ensures the study is safe and appropriate for everyone involved.
- Is your child aged 1 year up to 21 years old (not yet 22)?
- Has your child been diagnosed with chronic Graft Versus Host Disease (cGVHD)?
- Is your child able to take medicines as tablets?
- Are you able to attend regular follow-up appointments for your child?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If your child takes part in this study, they will receive the study medicine, ibrutinib, which comes as a tablet. They might also receive other medications like prednisone, which are also tablets. The doctors will carefully monitor your child's health throughout the study. This will likely involve regular hospital visits for check-ups, blood tests, and other assessments to see how the medicine is affecting them. They will be looking at how the medicine is handled by the body, checking for any side effects, and seeing if their cGVHD is getting better. They will also monitor your child's growth and development over time. The study has different parts, and how long your child participates may vary, but it will involve ongoing assessments and follow-ups over a period of time.
Potential risks and benefits
Locations (5)
- —France
- —Italy
- —Spain
- —Netherlands
- —Germany
Common questions
What is cGVHD?
cGVHD stands for chronic Graft Versus Host Disease. It's a complication that can happen after a stem cell transplant, where the transplanted cells fight against the patient's own body.
What is ibrutinib?
Ibrutinib is a medicine being tested in this study to treat cGVHD. It's also known by the brand name IMBRUVICA.
What does a 'Phase 1/2' study mean?
It means the study is in its early stages. Phase 1 focuses on finding the right dose and checking safety, while Phase 2 looks more at how well the medicine works and continues to monitor safety.
Will my child also take other medicines?
Yes, in addition to ibrutinib, your child might also be given prednisone, which is another medicine that comes in tablet form.
What if we decide not to continue in the study?
You are free to withdraw your child from the study at any time, for any reason, and this will not affect their regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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