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). The initial launch of the registry will be limited to patients residing in the United States; however, the registry may be extended to other regions and countries upon the applicable regulatory approvals. 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. Patients who meet all eligibility criteria will be able to participate. Patients will be required to read and sign an Institutional Review Board (IRB)-approved informed consent form prior to any registry-specific activity taking place.
At the time of informed consent, participants will be asked to indicate if they are interested in being contacted by registry personnel for potential participation in future clinical trials and/or studies. Participants who opt out will not be contacted for future studies.
No clinical procedures, testing, or diagnostics will be required by virtue of registry participation. Participants will enter relevant data into a web-based registry portal at regular intervals.
Participants will be asked to complete questionnaires related to their sVWD (including diagnosis, symptoms, treatments, family history, quality of life, etc.) at the time of enrollment and at regular follow up intervals. Questionnaires will be released into the registry in phases as modules for participant completion. The first phase (ie, registry launch) will include, at minimum, modules on demographics, medical history, and concomitant medications. Additionally, the Self-Administered Bleeding Assessment Tool (Self-BAT) will be completed by participants at baseline. Questionnaire modules and the Self-BAT may also be administered in person by qualified registry personnel (e.g., at annual VCF conferences). Laboratory and genetic sequencing data will be provided by the participant, if available. Participants who have not completed required questionnaires in the registry website within a 12-month period will be contacted by registry personnel for follow-up.
