(21824) A phase 3, single group treatment, open-label study to evaluate the safety of BAY 94-9027 infusions for prophylaxis and treatment of bleeding in previously treated children aged 7 to <12 years with severe hemophilia A
This research is a study looking into a new medicine called Jivi for treating severe haemophilia A in boys aged 7 to 11 who have already received treatment before. The main goal is to check how safe Jivi is, especially whether it causes any severe allergic reactions or if the body starts to fight against the medicine, making it less effective. Researchers are particularly interested in these reactions during the first four doses of Jivi. They will also be watching for any other bad reactions, checking if the body develops antibodies against the medicine, or if inhibitors (substances that stop the medicine from working) develop. The study will also look at how often bleeding episodes occur and how much Jivi is used.
At a glance
What is this study about?
This study is about a new treatment called Jivi for severe haemophilia A, a condition where blood doesn't clot properly. This condition mainly affects boys, and in severe cases, even small injuries can lead to serious bleeding. The aim of this study is to find out more about how safe Jivi is when used by boys aged 7 to 11 who have severe haemophilia A and have already received other treatments.
The researchers want to know if Jivi causes any strong allergic reactions or if the body starts to make substances called inhibitors that stop the medicine from working. They will be paying close attention to these things, especially during the first four times a child receives Jivi. This type of study is called a 'Phase 3' study, which means the medicine has already been tested in earlier stages, and now they are confirming its safety and how well it works on a larger group of people.
Besides checking for reactions and inhibitors, the study will also look at other important things. This includes any side effects the medicine might cause, how many bleeding episodes the children have, and how much of the medicine is needed to manage their condition. All this information will help doctors understand if Jivi is a good and safe option for children with severe haemophilia A.
Key takeaways
- This study is for boys with severe haemophilia A aged 7-11.
- It's testing a new medicine called Jivi to prevent and treat bleeding.
- The main focus is on the safety of Jivi, especially with the first four doses.
- Researchers will check for allergic reactions and if the body develops resistance.
- Participation involves receiving Jivi infusions and regular clinic visits.
Who may be eligible?
This study is specifically designed for boys with severe haemophilia A.
To join, a boy needs to be between 7 and 11 years old. He must also have severe haemophilia A, meaning his body produces less than 1% of the normal amount of a clotting factor called factor VIII. He should also have received other treatments for his haemophilia in the past.
Only boys can take part in this study. There might be other health requirements that the study doctors will discuss to make sure the study is safe and right for your child.
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 my child a boy?
- Is my child between 7 and 11 years old?
- Does my child have severe haemophilia A (less than 1% factor VIII)?
- Has my child received treatment for haemophilia A in the past?
What does participation involve?
If your child takes part in this study, he will receive the study medicine, Jivi, through an infusion (which is like a drip). The medicine comes as a powder that is mixed into a solution before being given.
The study is 'open-label', which means both you and the study team will know that your child is receiving Jivi. Your child will have regular visits to the clinic to receive the medication and for the study team to check on his health, take blood samples, and ask about any bleeds or side effects. The study will monitor your child for a period of time to track how well the medicine is working and if there are any problems.
Potential risks and benefits
Locations (2)
- —UnverifiedNorway
- —UnverifiedItaly
Common questions
What is severe haemophilia A?
It's a condition where a child's blood doesn't clot properly because they don't have enough of a special protein called factor VIII, leading to easy bruising and bleeding.
What is Jivi?
Jivi is the name of the new medicine being tested in this study, designed to help blood clot more effectively in people with haemophilia A.
What does 'open-label' mean?
It means everyone involved – you, your child, and the study doctors – will know that your child is receiving the study medicine, Jivi.
What are 'inhibitors'?
Inhibitors are substances the body can sometimes make that stop the haemophilia medicine from working properly, making treatment more difficult.
What does a 'Phase 3' study mean?
It means the medicine has already been checked for safety in earlier stages, and now it's being tested on a larger group to confirm it's safe and effective.
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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