A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)
This study is for adults and teenagers who have developed a serious condition called Thrombotic Microangiopathy (TMA) after a stem cell transplant. TMA is a rare but serious side effect that can happen after this type of transplant. The study is investigating a new medicine called Ultomiris (ravulizumab) to see if it can help people with TMA, improve their health, and prevent further problems. Researchers want to find out if Ultomiris is better than a dummy treatment (placebo) at helping people get better and stay well. Participants will be randomly assigned to receive either the study medicine or the dummy treatment, and doctors will closely monitor their health over several months.
At a glance
What is this study about?
Imagine you've had a stem cell transplant, a very intense treatment often used for certain cancers or blood disorders. Sometimes, a rare and serious complication called Thrombotic Microangiopathy (TMA) can develop afterwards. This condition affects your small blood vessels and can cause serious problems for your organs, like your kidneys.
This study is looking at a new medicine called Ultomiris. It's designed to help people who get TMA after their stem cell transplant. The aim is to see if Ultomiris can improve their health, help them recover, and reduce the chance of serious issues. Researchers want to compare Ultomiris with a 'dummy' treatment, called a placebo, to clearly see if the new medicine is effective. This is important because finding better ways to treat TMA could make a big difference for people after their transplant.
By taking part, you would be helping doctors understand if Ultomiris is a safe and effective treatment for TMA. This could lead to a new treatment option for many people in the future who face this complication. The study is carefully designed to make sure all participants are looked after and their health is closely monitored.
Key takeaways
- This study is for adults with TMA after a stem cell transplant.
- It tests a new medicine, Ultomiris, against a dummy treatment.
- Participation involves regular appointments and blood tests.
- The study aims to see if Ultomiris improves health and recovery.
- Your health will be closely monitored throughout.
- You can withdraw from the study at any time.
Who may be eligible?
This study is looking for adults aged 18 and older, of any sex, who have developed Thrombotic Microangiopathy (TMA) after having a stem cell transplant.
To be considered for this study, you must have recently had a stem cell transplant and doctors must have diagnosed you with TMA. Because this is a serious condition, there will be specific medical criteria your doctors will check to make sure the study is right for you, and that you can safely take part.
If you are interested, your specialist doctor will need to review all your medical information to see if you meet all the necessary requirements to join this study.
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 an adult aged 18 or older?
- Have you recently had a stem cell transplant?
- Have doctors diagnosed you with Thrombotic Microangiopathy (TMA) after your transplant?
- Are you able to attend regular study appointments?
- Are you willing to receive the study treatment as an infusion?
What does participation involve?
If you join this study, you will be randomly assigned to receive either the study medicine (Ultomiris) or a dummy treatment (placebo). Neither you nor your study doctors will know which one you are receiving. The treatment will be given as an infusion into your vein. This will happen regularly over a 26-week period.
You will have regular visits with the study team. These visits will involve blood tests to check your health and how the treatment is working. Doctors will also check for any side effects and how you are feeling. After the main 26-week treatment period, there will be follow-up visits to continue monitoring your health and see how you are doing in the longer term. The total duration of your involvement in the study could be several months, including the follow-up period.
Potential risks and benefits
Locations (9)
- —UnverifiedGermany
- —UnverifiedPoland
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedSweden
- —UnverifiedBelgium
- —UnverifiedItaly
- —UnverifiedNetherlands
- —UnverifiedGreece
Common questions
What is TMA?
TMA stands for Thrombotic Microangiopathy. It's a rare but serious complication that can happen after a stem cell transplant, affecting your small blood vessels.
What is a placebo?
A placebo is a 'dummy' treatment that looks like the real medicine but contains no active drug. It helps doctors see if the study medicine is truly effective compared to no treatment.
Will I know if I'm getting the real medicine?
No, this is a 'blinded' study, meaning neither you nor your doctors will know if you're receiving Ultomiris or the placebo. This helps ensure fair results.
How is the study medicine given?
The study medicine (or placebo) is given as an infusion, meaning it will be slowly delivered into one of your veins.
Can I leave the study if I change my mind?
Yes, you have the right to leave the study at any time, for any reason, without it affecting your usual medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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