A Multicentre, Open-Label Study Evaluating the Safety, Tolerability and Efficacy of MaaT013 in Ruxolitinib-Refractory or Intolerant Paediatric and Adolescent participants with Gastrointestinal Acute Graft-versus-Host Disease
This study is for children and teenagers who have a serious gut condition called acute Graft-versus-Host Disease (aGvHD). This can happen after a transplant when the new cells attack the patient's body. We're testing a new treatment, MaaT013, for those whose current medicine, ruxolitinib, isn't working or causing side effects. The main goals are to check how safe MaaT013 is and if patients can easily take it. We'll monitor for any side effects and see how well patients keep the medicine in their system. We'll also look at how much the medicine helps improve their gut GvHD over time, aiming for complete or good partial recovery.
At a glance
What is this study about?
This study is looking into a health problem called acute Graft-versus-Host Disease (aGvHD). This condition can occur in people, especially children and teenagers, after they've had a stem cell or bone marrow transplant. It happens when the new healthy cells from the donor start to see the patient's body as foreign and attack it. In this particular study, we are focusing on aGvHD that affects the gut, which can cause severe digestive problems.
The usual treatment for aGvHD might not always work for everyone, or it might cause unpleasant side effects. This study aims to find a new option, MaaT013, for young patients whose current medicine, ruxolitinib, isn't helping enough or is causing too many issues. We want to see if MaaT013 is a safer and more effective way to manage their gut symptoms.
During the study, doctors and nurses will carefully check how patients react to MaaT013. They will look for any side effects and how well the body handles the treatment. They will also measure if the treatment improves the aGvHD in the gut. The goal is to find medicines that can help young people feel better and live healthier lives after their transplants.
Key takeaways
- This study is testing a new treatment (MaaT013) for gut GvHD in young people.
- It's for those whose current medicine (ruxolitinib) isn't working or causing problems.
- The main goals are to check the new treatment's safety and effectiveness.
- You'll have regular check-ups and monitoring if you join.
- You can stop participating at any time.
- It could offer a new treatment option for gut GvHD.
Who may be eligible?
This study is looking for both male and female patients aged 18 years and older. Your doctor will need to check if your specific health situation matches the full list of study requirements.
Your doctor will discuss your medical history, current health, and previous treatments to make sure this study is the right fit for you. They will also need to confirm that you have been diagnosed with acute Graft-versus-Host Disease (aGvHD) in your gut and that your current treatment isn't working well enough or is causing problems.
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.
- Are you 18 years old or older?
- Have you been diagnosed with acute Graft-versus-Host Disease (aGvHD) in your gut?
- Is your current GvHD treatment (ruxolitinib) not working well or causing side effects?
- Are you prepared for regular hospital visits and check-ups related to the study?
What does participation involve?
If you decide to take part, you'll receive the study medicine, MaaT013, and possibly another medicine called Vancomycin. You'll need to visit the clinic regularly for check-ups. During these visits, the medical team will monitor your health, check your vital signs (like temperature and blood pressure), and take blood and other samples to see how you're responding to the treatment and if there are any side effects.
You'll also be asked to complete questionnaires to understand any stress or anxiety you might be feeling. The team will carefully watch how well you cope with taking the medicine and if there are any issues during the administration process. The study will involve regular follow-ups for at least 12 months, with close monitoring during the first 6 months. The total duration of your participation will depend on your response to the treatment and the study plan, which your doctor will explain fully.
Potential risks and benefits
Locations (4)
- —UnverifiedNetherlands
- —UnverifiedItaly
- —UnverifiedFrance
- —UnverifiedSpain
Common questions
What is Graft-versus-Host Disease (GvHD)?
GvHD is a condition that can happen after a transplant when the new cells from a donor start to attack the patient's body.
What does 'gut aGvHD' mean?
This means that the GvHD is specifically affecting your digestive system, like your stomach and intestines.
What is MaaT013?
MaaT013 is the name of the new medicine being tested in this study to treat gut GvHD.
What happens if I want to stop taking part?
You can choose to leave the study at any time without having to give a reason, and it won't affect your regular medical care.
Will I have to pay to be in the study?
No, all study-related treatments and tests are provided at no cost to you.
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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