The Severe Von Willebrand Disease (sVWD) Patient Registry

The Severe Von Willebrand Disease Patient Registry: A Longitudinal Natural History and Patient Outcomes Study

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.

Study Overview

• Status: Recruiting
• Conditions: VWD - Von Willebrand's Disease

Detailed Description

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.

• Study Type: Observational
• Enrollment (Estimated): 400

Contacts and Locations

• Study Contact: Name: Christina Morgenthaler, MS, MBA; Phone Number: (279) 346-6202; Email: morgenthaler@vwdregistry.org

Study Locations

• United States
  • Florida
    • Wellington, Florida, United States, 33414
      • Recruiting
      • VWD Connect Foundation
      • Contact: Christina Morgenthaler, MS, MBA; Phone Number: (279) 346-6202; Email: morgenthaler@vwdregistry.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Individuals diagnosed with severe VWD

Description

Inclusion Criteria:

1. Anyone meeting the diagnostic classification for von Willebrand disease

2. Any VWF antigen or activity:

   a. Result <20% regardless of bleeding phenotype, or b. Result <30% with excessive bleeding symptoms including: i. Bleeding that resulted in hospitalization, required surgical procedure, red blood cell transfusion, Hemoglobin decrease >2g/dL, or ii. Intracranial, intraspinal, pericardial, retroperitoneal, intramuscular bleeding with compartment syndrome, or iii. Persistent or recurrent bleeding that is disruptive of work or school.

3. Provide informed consent by participant or legally authorized representative
4. Be willing and able to comply with study procedures and data collection
5. Reside in the United States

NOTE: 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.

Exclusion Criteria:

1. Have a clinical diagnosis of acquired VWD

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Outcomes are not applicable; this study is a patient registry.	Statistical analyses will focus on simple characterization of the registry.	At least 5 years

Collaborators and Investigators

• Sponsor: VWD Connect Foundation
• Investigators: Principal Investigator: Christopher Walsh, MD, PhD, Mt. Sinai School of Medicine; Principal Investigator: Christina Morgenthaler, MS, MBA, VWD Connect Foundation; Principal Investigator: Mrinal Gounder, MD, Memorial Sloan Kettering Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): December 10, 2021
• Primary Completion (Estimated): December 1, 2031
• Study Completion (Estimated): December 1, 2031

Study Registration Dates

• First Submitted: June 21, 2022
• First Submitted That Met QC Criteria: June 24, 2022
• First Posted (Actual): June 29, 2022

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 17, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Von Willebrand Disease; 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; VWB; Von Willebrands Disease
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; von Willebrand Diseases; von Willebrand Disease, Type 3
• Other Study ID Numbers: VWD-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: De-identified/coded data requests: The researcher will request the types of research or preparation for research that will be conducted with the data as part of the application process. After the VCF MSAB subcommittee approves the scientific/technical merit of the request, the registry research team will export and provide applicable de-identified data to the researcher. No information that would directly link the data to the participants will be included in the output data.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No