Active clinical trials for "Von Willebrand Diseases"

In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well-controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.(7) The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US.

ATHN 9 is a natural history study to assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.

The working hypothesis is that patients with Von Willebrand Disease-type 2B may have neurological symptoms due to the chronic formation of platelets/Von Willebrand Factor aggregates. Interestingly, several patients present nonspecific neurological symptoms (nystagmus, headaches, memory disorder, …) which may be associated with cerebral microangiopathy and chronic microvessel inflammation secondary to Von Willebrand Disease-type 2B due to chronic exposure to Von Willebrand Factor-platelets complexes.

The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient data and guide clinical practice. With informed consent from the patient, the WBDR stores anonymous data about the person's disease, such as hemophilia type and severity, symptoms, and treatment.

The main aim of the study is to evaluate the effectiveness of prophylaxis with recombinant von Willebrand factor (rVWF) in children. This study will enroll those participants who have been previously treated with VWF product or with a plasma-derived VWF (pdVWF) product. In this study, participants will be treated with rVWF for 12 months. During the study, participants will visit the study clinic 6 times after treatment initiation.

The objectives of this study are to determine if transcutaneous auricular neurostimulation (tAN) can modulate hemostasis, improve perceived quality of life, and improve pain during the menstrual cycle of von Willebrand Disease (VWD) patients.

The goal of this interventional study is to test a hemostasis screening protocol and cardiac peri-procedural and post-operative hemostasis pathway to improving bleeding complications and improve patient survival for children with Alagille syndrome and complex cardiac conditions. The main questions it aims to answer are: Are children with Alagille syndrome with cardiac anomalies more likely to have acquired von Willebrand syndrome (a condition that causes increased bleeding) Does implementation of a novel screening protocol to detect pre-operative bleeding conditions decrease intra-operative and/or post-operative bleeding complications and mortality risk? Does implementation of a novel screening protocol to detect and treat bleeding conditions cause thrombotic complications? Participants will undergo additional hematology and bleeding disorder screening prior to cardiac surgery. They will additionally undergo a detailed family screening for a history of bleeding by a genetic counselor. Researchers will compare these findings with children who have similar complex cardiac conditions requiring surgery, but who do not have Alagille syndrome to see if bleeding conditions and complications are more or less common in children with Alagille syndrome.

A web-based registry will be created by the sponsor, VWD Connect Foundation (VCF), to collect data on patients with severe Von Willebrand Disease (sVWD). Data will be self-reported by patients and/or collected by registry personnel, as appropriate. The purpose of the sVWD Patient Registry is to create a database of well-characterized (with respect to demographics, medical history, symptoms, laboratory and genetic data, etc.) patients with sVWD for participation in retrospective and prospective research.

The main aim of this study is to describe the experience and unmet needs of persons living with VWD and their caregivers in Canada. The survey is planned to be done in two phases: The first phase will be directed at adult participants; the second phase will focus on children and teenagers. At the end of the first phase the Sponsor will decide if the second phase will be started. Participants and their caregivers will be asked to answer a set of questions either using an online questionnaire or through interviews. The participant/caregiver's perception, experience, satisfaction, and unmet needs, and need for new treatments or new indications will be determined based on their responses to the questions.

The objective of this observational study HOPSCOTcH-WILL II is to provide an accurate and detailed account of current Von Willebrand Disease (VWD) therapeutic management by collecting real-life data on hemorrhagic treated events in the 5 Western French Hemophilia Treatment Center. The HOPSCOTCH-WILL II study aims to describe therapeutic management of patients with VWD, following the provision of a recombinant drug in France; It will also permit to carry out a budget impact analysis to quantify the economic significance of this new era.