Ongoing Clinical Trials for Von Willebrand’s Disease
Von Willebrand’s disease is a genetic bleeding disorder that affects blood clotting. Currently, there are 4 ongoing clinical trials testing different treatment approaches for various types of this condition, including severe forms and Type 2B and Type 3 variants. These studies are taking place across multiple European countries and are evaluating medications designed to prevent bleeding episodes and improve quality of life for both children and adults with the condition.
Clinical trial locations
- Austria
- Belgium
- France
- Study on Preventing Bleeding in Children with Severe von Willebrand Disease Using Vonicog Alfa and Octocog Alfa
- Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery
- Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease
- Germany
- Ireland
- Italy
- Study on Preventing Bleeding in Children with Severe von Willebrand Disease Using Vonicog Alfa and Octocog Alfa
- Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery
- Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease
- Netherlands
- Poland
- Spain
- Study on Preventing Bleeding in Children with Severe von Willebrand Disease Using Vonicog Alfa and Octocog Alfa
- Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery
- Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease
- Sweden
Study on Preventing Bleeding in Children with Severe von Willebrand Disease Using Vonicog Alfa and Octocog Alfa
This trial is testing a preventive treatment approach for children with severe von Willebrand disease. The condition causes a deficiency in a protein called von Willebrand factor, which is essential for proper blood clotting. Without enough of this protein, children can experience frequent and excessive bleeding episodes.
Main focus: The study evaluates whether regular treatment with recombinant von Willebrand factor can effectively reduce bleeding episodes in children compared to their bleeding history before joining the study. The treatment is given through intravenous injection and aims to replace the missing or defective protein that helps blood to clot.
Who can participate: Children with confirmed severe von Willebrand disease, shown by specific blood test results indicating very low levels of the clotting protein. Participants must have experienced bleeding episodes in the past and be willing to follow the study procedures for up to 12 months.
Who cannot participate: Children with a history of severe allergic reactions to the study medication, those who have recently taken other investigational drugs, pregnant or breastfeeding participants, and those with medical conditions that would make participation unsafe or difficult to complete the study procedures.
Investigational treatment: The study uses recombinant von Willebrand factor (rVWF), a laboratory-made version of the natural clotting protein. This medication is administered regularly to prevent bleeding episodes rather than treating them after they occur.
Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery
This clinical trial examines how well recombinant von Willebrand factor works for managing bleeding episodes in children under 18 years old with severe von Willebrand disease. The treatment may be used alone or combined with another medication called ADVATE, which provides factor VIII, another important clotting protein.
Main focus: The study measures how effectively the treatment controls bleeding events using a standardized 4-point scale. It also evaluates safety, tolerability, and how the body processes the medication. The treatment is designed to help during both unexpected bleeding episodes and planned or emergency surgeries.
Who can participate: Children younger than 18 with diagnosed severe von Willebrand disease, showing very low blood levels of von Willebrand factor. For previously treated children, they must have experienced at least one bleeding event in the past year requiring treatment. Both the child and their parent or guardian must agree to participate and follow study requirements.
Who cannot participate: Children 18 years or older, those with other medical conditions that might interfere with the study, participants unable to attend required visits or follow procedures, those planning major surgery, and children currently enrolled in another clinical trial.
Investigational treatments: The trial uses recombinant von Willebrand factor (rVWF) either alone or combined with ADVATE, which provides factor VIII. Together, these medications aim to improve blood clotting and control bleeding episodes more effectively.
Study on the Effects of BT200 (Rondaptivon Pegol) for Patients with Type 2B von Willebrand Disease
This study focuses on a specific variant called Type 2B von Willebrand disease, where the von Willebrand factor protein has increased binding to platelets. This abnormal interaction causes platelets to clump together in the bloodstream, reducing the overall platelet count and leading to bleeding episodes.
Main focus: The trial evaluates how BT200 (rondaptivon pegol) affects platelet count and reduces bleeding events over a four-week treatment period. The medication is given as an injection under the skin and aims to improve how blood components work together for proper clotting.
Who can participate: Adults aged 18 years or older with confirmed Type 2B von Willebrand disease who have low platelet counts and a recent history of bleeding. Participants must be able to understand the study, provide informed consent, and cooperate with study procedures.
Who cannot participate: Patients who fall into vulnerable populations or who do not meet the specific age and diagnostic criteria for Type 2B von Willebrand disease.
Investigational treatment: BT200 (rondaptivon pegol) works by interacting with blood components involved in clotting to improve their function. The medication is designed to increase platelet numbers and reduce bleeding episodes in patients with this specific type of the disease.
Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease
This trial studies emicizumab as a preventive treatment for Type 3 von Willebrand disease, the most severe form of the condition. Patients with Type 3 have very low or undetectable levels of von Willebrand factor and often low levels of factor VIII as well, leading to frequent and severe bleeding episodes.
Main focus: The study compares regular preventive treatment with emicizumab to the standard approach of treating bleeding only when it occurs. Researchers will monitor the number of bleeding episodes over time, assess safety, and evaluate the medication’s long-term effects on participants’ health and quality of life.
Who can participate: Patients at least 2 years old with confirmed Type 3 von Willebrand disease based on specific blood tests. Participants must have adequate blood function and a history of using both on-demand and preventive treatments. Those able to have children must agree to use contraception during the study.
Who cannot participate: Patients with other bleeding disorders besides Type 3, those with recent or planned major surgery, participants currently in another trial, anyone with known allergies to emicizumab, those with severe liver or kidney disease, pregnant or breastfeeding women, and patients unable to comply with study procedures.
Investigational treatment: Emicizumab is a monoclonal antibody given as an injection under the skin on a regular schedule. It works by mimicking a protein that helps blood to clot, thereby reducing the risk of bleeding episodes. The medication is designed to provide continuous protection rather than treating bleeding after it starts.
Summary
The four ongoing clinical trials for von Willebrand disease reflect the diversity of this genetic bleeding disorder. Two trials focus specifically on children with severe forms of the disease, testing recombinant von Willebrand factor either alone or in combination with factor VIII. These pediatric studies emphasize preventive treatment approaches and management during both bleeding episodes and surgical procedures.
The remaining two trials target specific variants of the disease in broader age groups. One examines Type 2B von Willebrand disease in adults, while another studies Type 3, the most severe form, in patients aged 2 years and older. These trials test different therapeutic approaches, including BT200 and emicizumab, representing novel mechanisms for managing this challenging condition.
Geographically, the trials show strong representation across Western Europe, with France, Italy, and Spain participating in three trials each. Belgium, Austria, and several other European countries are also actively involved. This distribution reflects the collaborative nature of rare disease research and provides access to trials for patients across multiple countries.
