Efficacy and Safety of BT200 (rondaptivon pegol) in Patients with Type 2B von Willebrand disease
Type 2B von Willebrand disease is a rare, inherited bleeding condition. People with this condition often have low numbers of platelets, which are tiny cells needed to help the blood clot. This can lead to frequent nosebleeds, easy bruising, and heavy bleeding after minor injuries or surgery. This Phase II clinical trial looks at an experimental drug called rondaptivon pegol (BT200). Researchers are comparing it with a harmless saltwater solution (saline) to find out how safe it is and how well it works. The main goals are to check whether the treatment can safely boost platelet counts and cut down the number of bleeding events patients experience each month. The study will also measure how the medicine moves through the body and how it affects blood clotting over time.
At a glance
What is this study about?
Type 2B von Willebrand disease is a rare, lifelong blood condition. Normally, when you get a cut, a special protein called von Willebrand factor works together with tiny blood cells called platelets to form a plug and stop the bleeding. In type 2B, this protein is overactive and sticks to platelets too easily. This causes platelets to be removed from the blood too quickly, leaving people with low platelet counts and an increased risk of troublesome bleeds.
Current treatments do not always fully solve the problem of low platelets. This trial is investigating a new medicine known as rondaptivon pegol (or BT200). The treatment is designed to calm down the overactive clotting protein. By doing this, doctors hope it will prevent platelets from being cleared away unnecessarily, allowing them to do their normal job when an injury happens.
Finding a treatment that protects platelets could make a real difference to daily life. If successful, this medicine could give people with type 2B von Willebrand disease better protection against unexpected bleeding, reduce the need for emergency hospital visits, and offer a more targeted way to manage the condition.
Key takeaways
- Testing a new medicine called rondaptivon pegol (BT200) for type 2B von Willebrand disease.
- The main goals are to increase platelet levels and lower the number of bleeding episodes.
- Open to adults aged 18 and over with a confirmed diagnosis.
- Involves clinic visits, regular blood tests, and keeping a diary of any bleeds.
- Taking part is voluntary, and you can withdraw at any point.
Who may be eligible?
This clinical trial is designed specifically for adults who have a confirmed diagnosis of type 2B von Willebrand disease. Both men and women aged 18 and older may be able to take part.
Because this is an exploratory study, the doctors running the trial will need to check your general health and review your medical history. They will check your baseline platelet counts and ensure it is safe for you to try a new medicine.
People who are pregnant, breastfeeding, or who have certain other serious medical conditions may not be eligible to join. Your hospital doctor or the study team can go through the full medical criteria with you to see if the trial is suitable.
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.
- I am 18 years of age or older.
- I have been diagnosed with type 2B von Willebrand disease.
- I experience frequent bleeds or have low platelet counts.
- I am able and willing to attend regular hospital appointments.
- I am happy to keep a diary of my symptoms and bleeding events.
What does participation involve?
Taking part in this study means visiting a specialist hospital clinic on a regular schedule. During these visits, doctors and nurses will closely monitor your health. You will receive either the study medicine (rondaptivon pegol) or an inactive saltwater solution (placebo). You will have routine physical check-ups and regular blood tests to check your platelet numbers and see how your body breaks down the medicine. You will also be asked to keep track of any bleeding events you experience in a daily diary. The study team will explain the exact number of visits and how many months your participation will last before you agree to join.
Potential risks and benefits
Locations (1)
- —UnverifiedAustria
Common questions
What is the difference between this trial medicine and my current treatment?
Standard treatments often replace clotting factors temporarily, whereas this trial drug aims to specifically stop platelets from being clumped together and destroyed too early.
Will I definitely receive the new medicine?
Not necessarily. The study compares the new medicine against a dummy saltwater injection (placebo). Neither you nor your doctor may know which one you receive at first.
Will taking part cost me anything?
No, the study medicine and all trial-related tests are provided free of charge. Studies usually help cover reasonable travel expenses to and from clinic visits.
Can I leave the study if I change my mind?
Yes. You are free to stop participating at any time without giving a reason, and your normal healthcare will continue as usual.
How will my bleeding be monitored?
You will be asked to record any bleeding episodes, like nosebleeds or gum bleeds, in a diary, and the medical team will review this at your check-ups.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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