Registry for patients with severe Von Willebrand Disease
The Severe Von Willebrand Disease Patient Registry: A Longitudinal Natural History and Patient Outcomes Study
This study is creating a database to gather information from people with severe Von Willebrand Disease to help improve future research and treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 400 (estimated) |
| Sex | All |
| Sponsor | VWD Connect Foundation Academic / other |
| Locations | 1 site (Wellington, Florida) |
| Trial ID | NCT05437536 on ClinicalTrials.gov |
What this trial studies
This registry aims to collect comprehensive data on patients diagnosed with severe Von Willebrand Disease (sVWD) through a web-based platform. Participants will self-report their medical history, symptoms, and laboratory data, which will be used to create a detailed database for future research. Initially, the registry will focus on patients residing in the United States, with potential expansion to other regions. Participants will provide informed consent and may be contacted for future studies based on their interest.
Who should consider this trial
Good fit: Ideal candidates for this registry are individuals with a clinical diagnosis of Von Willebrand Disease and Von Willebrand factor levels below 20%.
Not a fit: Patients with acquired Von Willebrand Disease will not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could enhance understanding of severe Von Willebrand Disease and facilitate the development of targeted treatments.
How similar studies have performed: While registries for rare diseases have shown success in gathering valuable data, this specific approach for sVWD is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Wellington, Florida
- VWD Connect Foundation — Wellington, Florida, United States (Recruiting)
Study contacts
- Principal investigator: Christopher Walsh, MD, PhD — Mt. Sinai School of Medicine
- Study coordinator: Christina Morgenthaler, MS, MBA
- Email: morgenthaler@vwdregistry.org
- Phone: (279) 346-6202
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.