[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05437536":3,"trial-entities:NCT05437536":83,"trial-summary:NCT05437536":88},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":33,"primary_purpose":34,"phases":35,"enrollment_info":36,"interventions":39,"primary_outcomes":40,"secondary_outcomes":45,"sex":46,"minimum_age":34,"maximum_age":34,"healthy_volunteers":15,"eligibility_criteria":47,"std_ages":51,"locations":55,"central_contacts":70,"overall_officials":72,"references":81,"see_also_links":82},"NCT05437536","VWD-001","The Severe Von Willebrand Disease (sVWD) Patient Registry","The Severe Von Willebrand Disease Patient Registry: A Longitudinal Natural History and Patient Outcomes Study","RECRUITING","2031-12","2026-04","2026-04-22","2021-12-10","VWD Connect Foundation","OTHER",false,"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\u002For 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.","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\u002For 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.\n\nAt 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\u002For studies. Participants who opt out will not be contacted for future studies.\n\nNo 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.\n\nParticipants 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.",[19],"VWD - Von Willebrand's Disease",[21,22,23,24,25,26,27,28,29,30,31,32],"VWD","Severe VWD","VWD Type 1 Severe","VWD Type 1C","VWD Type 2A","VWD Type 2B Severe","VWD Type 2M Severe","VWD Type 2N Severe","VWD Type 3","Von Willebrand Disease","VWB","Von Willebrands Disease","OBSERVATIONAL",null,[],{"count":37,"type":38},400,"ESTIMATED",[],[41],{"measure":42,"description":43,"timeFrame":44},"Outcomes are not applicable; this study is a patient registry.","Statistical analyses will focus on simple characterization of the registry.","At least 5 years",[],"ALL",{"inclusion":48,"exclusion":49,"raw_text":50},[],[],"Inclusion Criteria:\n\n1. Anyone meeting the diagnostic classification for von Willebrand disease\n2. Any VWF antigen or activity:\n\n   a. Result \\\u003C20% regardless of bleeding phenotype, or b. Result \\\u003C30% with excessive bleeding symptoms including: i. Bleeding that resulted in hospitalization, required surgical procedure, red blood cell transfusion, Hemoglobin decrease \\>2g\u002FdL, or ii. Intracranial, intraspinal, pericardial, retroperitoneal, intramuscular bleeding with compartment syndrome, or iii. Persistent or recurrent bleeding that is disruptive of work or school.\n3. Provide informed consent by participant or legally authorized representative\n4. Be willing and able to comply with study procedures and data collection\n5. Reside in the United States\n\nNOTE: For inclusion criteria 1 and 2, it is not expected that participants will submit evidence of clinical diagnosis at screening; a participant's own confirmation that they have severe VWD will be sufficient for meeting the enrollment requirement and obtaining access to the registry. Laboratory values will be provided during the conduct of the study, and participants with VWF antigen or activity not meeting inclusion criterion 2 may be excluded from data analysis on a case-by-case basis in consultation with a subcommittee of the VWD Connect Foundation Medical and Scientific Advisory Board.\n\nExclusion Criteria:\n\n1\\. Have a clinical diagnosis of acquired VWD",[52,53,54],"CHILD","ADULT","OLDER_ADULT",[56],{"facility":13,"status":8,"city":57,"state":58,"zip":59,"country":60,"contacts":61,"geoPoint":67},"Wellington","Florida","33414","United States",[62],{"name":63,"role":64,"phone":65,"email":66},"Christina Morgenthaler, MS, MBA","CONTACT","(279) 346-6202","morgenthaler@vwdregistry.org",{"lat":68,"lon":69},26.65868,-80.24144,[71],{"name":63,"role":64,"phone":65,"email":66},[73,77,80],{"name":74,"affiliation":75,"role":76},"Christopher Walsh, MD, PhD","Mt. Sinai School of Medicine","PRINCIPAL_INVESTIGATOR",{"name":78,"affiliation":79,"role":76},"Mrinal Gounder, MD","Memorial Sloan Kettering Cancer Center",{"name":63,"affiliation":13,"role":76},[],[],{"nct_id":4,"conditions":84,"biomarkers":86},[85],"von Willebrand Disease",[87],"Von Willebrand Factor Human",{"nct_id":4,"found":89,"summary":90,"prompt_version":100},true,{"design":91,"status":92,"heading":93,"summary":94,"follow_up":95,"word_count":96,"commitments":97,"compensation":98,"drugs_mentioned":99},"This is an observational study, meaning no interventions are given. It aims to enroll 400 participants.","completed","Severe Von Willebrand Disease (sVWD) Patient Registry","This is a patient registry for individuals with severe Von Willebrand Disease (sVWD), a bleeding disorder. The VWD Connect Foundation is creating a web-based database to collect information about people with sVWD, including their health history, symptoms, and lab results. This information will help researchers better understand sVWD and identify people who might be interested in future studies. You can join if you meet the diagnostic criteria for von Willebrand disease, specifically if your VWF antigen or activity is less than 20%, or less than 30% with significant bleeding symptoms that required hospitalization, surgery, blood transfusions, or a large drop in hemoglobin. The goal is to gather data for at least 5 years to support future research.","Outcomes are measured for at least 5 years, indicating a long-term follow-up for data collection.",117,"You will provide self-reported data or have registry personnel collect it. No clinical procedures, testing, or diagnostics are required for participation.","Not stated in the trial record.",[],"v2"]