A Prospective Screening Study to Track Bleeding Events and Treatments in People with Von Willebrand Disease

What this Trial Is About

Researchers are studying people with Von Willebrand Disease VWD, focusing on how often bleeding events happen, their impact on quality of life, and the social and clinical effects of bleeds. This screening study gathers baseline data on bleeding and treatment rates in participants with VWD, mainly Type 1, but may include Types 2 and 3 with sponsor approval. The findings will help compare outcomes in future clinical studies like Velora Pioneer. The study involves screening and a baseline check, followed by an observation period of about 4 months during which participants will have telemedicine check-ins every two weeks. These check-ins monitor bleeding events and treatment use through a bleed diary. Participants can opt to extend this observation period up to 12 months if they wish. Participants will provide information about their bleeding events and treatments during the study. Researchers will assess bleeding rates, hospital stays, and quality of life using questionnaires and clinical evaluations. Outcomes measured include annual bleeding rates, treatment use, iron status, and bleeding severity scores. The study concludes with data collection over the observation period, helping to understand the experience of people with VWD over time.

A Study of Bleeding and Treatment in Participants With Von Willebrand Disease