A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to <18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)
This study is for children and teenagers, from one month old up to 17 years old, who have developed a serious condition called Thrombotic Microangiopathy (TMA) after having a stem cell transplant. TMA can cause problems in different parts of the body. The study is testing a medicine called Ultomiris. Doctors want to see how well Ultomiris works to treat TMA in these young patients. They will be looking at whether it helps improve the TMA, how quickly it works, and how it affects different organs like the kidneys, heart, and lungs. They will also be monitoring the children's overall health and well-being during the study.
At a glance
What is this study about?
This study is looking at a condition called Thrombotic Microangiopathy (TMA) that can sometimes happen after a special type of treatment called a stem cell transplant. Stem cell transplants are used to treat serious illnesses, but sometimes they can lead to complications like TMA, where tiny blood vessels get damaged. This can affect organs throughout the body.
Doctors are testing a medicine called Ultomiris to see if it can help children and teenagers who get TMA after their stem cell transplant. This is a "Phase 3" study, which means it's one of the final stages of testing a new medicine before it might be made more widely available. The main goal is to find out if Ultomiris can successfully treat TMA.
Beyond just treating TMA, the study will also look at other important things. For example, they'll check how quickly Ultomiris starts to work, and if it helps improve problems with organs like the kidneys, heart, lungs, brain, and digestive system over six months and a year. They'll also monitor if TMA comes back, how many patients survive, and other blood-related measures to get a full picture of the medicine's effects.
Key takeaways
- This study is for children (1 month to under 18) with TMA after a stem cell transplant.
- It's testing a medicine called Ultomiris to see if it helps treat TMA.
- The study will check how Ultomiris affects TMA, organ function, and overall health.
- Participants will receive the study medicine via infusion, along with their usual care.
- Regular check-ups and monitoring will be part of taking part.
- You can withdraw your child from the study at any time.
Who may be eligible?
This study is specifically for young patients. To be considered, a child must be at least one month old but not yet 18 years old. Both boys and girls are able to take part.
The most important requirement is that the child must have developed Thrombotic Microangiopathy (TMA) after recently having a stem cell transplant. This means the study is only for those who have this particular complication.
There will be other detailed medical checks to make sure that taking part in the study is safe and appropriate for each child. A doctor will carefully review all the child's medical information to determine if they meet all the necessary criteria.
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 between 1 month and under 18 years old?
- Has your child recently had a stem cell transplant?
- Has your child been diagnosed with Thrombotic Microangiopathy (TMA) after the transplant?
- Is your child prepared to receive the study medicine as an infusion?
- Are you able to attend regular hospital visits for assessments and treatments?
What does participation involve?
If your child is eligible and you decide to take part, they will receive the study medicine, Ultomiris, as an infusion (meaning it's given slowly into a vein). This medicine will be given alongside their usual care, often called 'best supportive care', which means they'll still receive all the necessary treatments and support for their overall health.
Throughout the study, your child will have regular check-ups and assessments. These will involve doctors carefully monitoring their TMA, checking how their organs are functioning (such as kidneys, heart, and lungs), and taking blood samples. These checks help the medical team understand how the medicine is working and how your child is feeling. They will also monitor for any side effects.
The study will continue for a period of time, with assessments at six months and a year, to see the long-term effects of the medicine and ensure your child is doing well. The total duration of participation will be explained by the study team.
Potential risks and benefits
Locations (4)
- —UnverifiedSpain
- —UnverifiedFrance
- —UnverifiedGermany
- —UnverifiedItaly
Common questions
What is a stem cell transplant?
A stem cell transplant is a medical procedure that replaces unhealthy blood-forming cells with healthy cells, often used to treat certain cancers and blood disorders.
What is Thrombotic Microangiopathy (TMA)?
TMA is a serious condition where tiny blood vessels get damaged, which can lead to problems with blood clotting and affect organs like the kidneys, brain, and heart.
What is Ultomiris?
Ultomiris is the name of the medicine being tested in this study. It's given by infusion to help treat TMA.
Will my child still get their usual care?
Yes, kids in this study will still receive their standard, best possible care alongside the study medicine.
How long will my child need to be in the study?
Your child will be monitored for at least a year, with assessments at six months and twelve months, to see how the medicine works over time.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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