A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
This study is investigating a new medicine called Vonicog Alfa for children with severe Von Willebrand Disease (VWD). VWD is a condition that affects how your blood clots. The main goal is to see how well Vonicog Alfa prevents bleeding when given regularly. This is for children who have already been treated with other VWD medicines. Participants will take Vonicog Alfa for a year and visit the clinic five times during that period. This study aims to find a potentially better way to manage bleeding for children living with this condition.
At a glance
What is this study about?
This study is designed to learn more about a new medicine called Vonicog Alfa. It's for children who have a serious blood condition called Von Willebrand Disease (VWD). People with VWD have trouble with their blood clotting properly, which can lead to bleeding problems.
The main purpose of this study is to see if Vonicog Alfa can help prevent bleeding episodes in children. This type of treatment, where medicine is given regularly to prevent problems, is called prophylaxis. The study is particularly interested in children who have already been using other medicines for their VWD, either to stop bleeding when it happens or as a regular preventative treatment.
By taking part, children will receive Vonicog Alfa for 12 months. Researchers will then carefully check how effective it is at reducing bleeding events. This research is important because it could lead to new or improved ways to help children manage their VWD and live healthier lives.
Key takeaways
- This study is testing a new medicine, Vonicog Alfa, for severe Von Willebrand Disease.
- It aims to see if the medicine can prevent bleeding in children.
- Children aged under 18 who have used other VWD treatments are eligible.
- Participants will receive the medicine for one year.
- There will be 5 clinic visits over the year to check on progress.
Who may be eligible?
To be considered for this study, children need to have a confirmed diagnosis of severe Von Willebrand Disease. This means their blood clotting factor levels are quite low, and they've needed treatment for bleeding in the past. We're looking for children who are under 18 years old.
Also, a child must have received other Von Willebrand Factor (VWF) treatments previously. This could be either regular treatment to prevent bleeding for at least 12 months (or 6 months if under 2 years old) or treatment given only when bleeding happened, for at least 12 months (or 6 months if under 2 years old).
Finally, for older children (2 years and above), we need to be able to look at their medical records for the past year to understand their bleeding history and how it was treated. For those under 2 years old, we'll need similar records from the past 6 months.
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.
- Does your child have a diagnosis of severe Von Willebrand Disease?
- Is your child under 18 years old?
- Has your child been treated for Von Willebrand Disease before?
- Do you have good records of your child's bleeding history for the past 6-12 months?
- Has a doctor recommended preventative treatment for your child's VWD?
What does participation involve?
If your child takes part in this study, they will receive the study medicine, Vonicog Alfa, for 12 months. After they start the treatment, they will need to visit the study clinic five times over that year for checks. These visits will help the doctors see how your child is doing and how the medicine is working. The total study duration for each child will be 12 months for receiving the medicine, plus the time for these clinic visits.
Potential risks and benefits
Locations (21)
- University of Alabama at BirminghamVerified postcodeBirmingham, United States· Recruiting
- Bleeding and Clotting Disorders InstituteVerified postcodePeoria, United States· Recruiting
- Riley Hospital for Children Indiana University HealthVerified postcodeIndianapolis, United States· Recruiting
- University of Iowa Hospitals & Clinics PARENTVerified postcodeIowa City, United States· Recruiting
- Childrens Hospital of MichiganVerified postcodeDetroit, United States· Recruiting
- Michigan State University Center for Bleeding Disorders & Clotting DisordersVerified postcodeEast Lansing, United States· Recruiting
- Children's Health Care d/b/a Children's MinnesotaVerified postcodeMinneapolis, United States· Recruiting
- Cure 4 the KidsVerified postcodeLas Vegas, United States· Recruiting
- Rutgers - Robert Wood Johnson Medical SchoolVerified postcodeNew Brunswick, United States· Recruiting
- New York - Presbyterian/Weill Cornell Medical CenterVerified postcodeNew York, United States· Recruiting
- Medical University of South Carolina (MUSC)Verified postcodeCharleston, United States· Recruiting
- Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419Verified postcodeLille, France· Recruiting
Common questions
What is Von Willebrand Disease?
It's a condition where your blood doesn't clot properly, which can lead to bleeding problems.
What is Vonicog Alfa?
It's a new medicine being studied to help prevent bleeding in children with Von Willebrand Disease.
How long will my child take part in the study?
Your child will receive the study medicine for 12 months and have 5 clinic visits during that time.
Will my child stop their current VWD medicine?
The study is for children who have used VWD medicines before, and the doctor will discuss whether switching to Vonicog Alfa is suitable.
Are there any age limits for this study?
Yes, children under 18 years old can take part.
How to find out more
Takeda Contact
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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