A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease
This study is for children with a serious bleeding disorder called severe von Willebrand Disease. Doctors want to see how well a new medicine, VEYVONDI, works to stop bleeding episodes and if it's safe. They also want to understand how it behaves in the body. The study will look at giving VEYVONDI on its own, or sometimes with another medicine called ADVATE, for everyday bleeding and also during planned or emergency operations. The main goal is to check if VEYVONDI successfully stops bleeding. They will also keep a close eye on any side effects and how the medicine moves through the body. This is a Phase 3 study, which means it’s a big step towards potentially making the medicine widely available.
At a glance
What is this study about?
This study is looking into a new treatment called VEYVONDI for children who have severe von Willebrand Disease. This is a condition where a child's blood doesn't clot properly, leading to bleeding that can be hard to control. The researchers want to understand how effective VEYVONDI is at stopping bleeding episodes, whether it's safe to use, and how it affects the body itself.
The study will observe children when they experience bleeding and after they've been given VEYVONDI. Sometimes, VEYVONDI will be given alone, and other times it might be given alongside another medicine called ADVATE. They also want to see how well VEYVONDI works during different types of surgery, both planned operations and emergency ones. By doing this, they hope to get a full picture of how useful and reliable VEYVONDI could be for children with this condition. This kind of research is very important because it helps doctors find better ways to manage serious conditions like von Willebrand Disease.
This is a 'Phase 3' study, which means it's one of the final steps before a new medicine might be approved for wider use. It involves a larger group of patients than earlier studies and confirms the findings about how well the medicine works and its safety. The information gathered will be crucial in deciding if VEYVONDI can help children live healthier lives with fewer bleeding problems.
Key takeaways
- The study assesses a new medicine, VEYVONDI, for children with severe von Willebrand Disease.
- It aims to see how well VEYVONDI stops bleeding and its safety, including during surgery.
- Children will receive VEYVONDI, sometimes with ADVATE, as treatment.
- This is a Phase 3 study, a key step before potential wider availability.
- Close monitoring for side effects and how the medicine works in the body will occur.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is specifically designed for children who have been diagnosed with severe hereditary von Willebrand Disease. We believe all children diagnosed with this specific condition should be able to take part if their doctor feels it's right.
There aren't any specific age limits mentioned, meaning that if a child has severe hereditary von Wille Willebrand Disease, their doctor should be able to assess if the study is suitable for them. Both boys and girls can take part. Your doctor will carefully review your child's medical history to make sure they meet all the specific requirements for joining the 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.
- My child has been diagnosed with severe hereditary von Willebrand Disease.
- My child is a boy or a girl.
- My child's doctors agree that their overall health makes them suitable for a clinical study.
- I am comfortable with my child receiving study medication and having regular checks and blood tests.
What does participation involve?
If your child participates, they will receive either VEYVONDI (in two different strengths) or sometimes ADVATE, as a powder that's mixed into a solution and given as an injection. The study will monitor how well these medicines stop bleeding episodes and how your child reacts. This includes checking for any side effects. For children undergoing surgery, the medicine's effectiveness will be assessed up to 14 days after their last dose around the time of surgery. The study will involve regular visits to the clinic so doctors can take blood samples to see how the medicine is working in the body and check on your child's health. The total length of your child's participation will depend on the study plan, but it's designed to gather enough information about the medicine's long-term effects and safety.
Potential risks and benefits
Locations (5)
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedAustria
- —UnverifiedBelgium
- —UnverifiedItaly
Common questions
What is von Willebrand Disease?
It's a bleeding disorder where the blood doesn't clot properly, leading to longer or heavier bleeding than usual.
What is VEYVONDI?
VEYVONDI is a new medicine being studied to help improve blood clotting for children with severe von Willebrand Disease.
Will my child definitely get the new medicine?
Yes, all children in this study will receive VEYVONDI, sometimes on its own, and sometimes with ADVATE, as part of their treatment.
What does a 'Phase 3' study mean?
Phase 3 means this is one of the final stages of testing a new medicine, involving more people to confirm its effectiveness and safety before it can be approved.
Can we stop participating at any time?
Yes, you are completely free to withdraw your child from the study at any point without any impact on their normal 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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