VWD - Von Willebrand's DiseaseNCT05437536
The Severe Von Willebrand Disease (sVWD) Patient Registry
An observational study of VWD - Von Willebrand's Disease, run by VWD Connect Foundation at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- VWD - Von Willebrand's Disease
- How many people
- 400 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Dec 2021to Dec 2031
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals diagnosed with severe VWD
Who may be able to join
- Anyone meeting the diagnostic classification for von Willebrand disease
- Any VWF antigen or activity:
- 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.
- Provide informed consent by participant or legally authorized representative
- Be willing and able to comply with study procedures and data collection
- 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.
Who cannot join
- 1. Have a clinical diagnosis of acquired VWD
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
VWD Connect Foundation
Wellington, Florida, 33414
Christina Morgenthaler, MS, MBA, (279) 346-6202, morgenthaler@vwdregistry.org
About this study
Official title: 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.
Read the 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.
- Conditions
- VWD - Von Willebrand's Disease
- Keywords
- 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
- Medical subject headings
- von Willebrand Diseases, von Willebrand Disease, Type 3
What is being measured
The primary outcome is the main question the study is designed to answer.
Outcomes are not applicable; this study is a patient registry.
Statistical analyses will focus on simple characterization of the registry.
Measured: At least 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2022
- Study start10 Dec 2021
- Primary completionDec 2031 (estimated)
- Study completionDec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VWD - Von Willebrand's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 400 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- VWD Connect Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christopher Walsh, MD, PhD, Principal investigator, Mt. Sinai School of MedicineMrinal Gounder, MD, Principal investigator, Memorial Sloan Kettering Cancer CenterChristina Morgenthaler, MS, MBA, Principal investigator, VWD Connect Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05437536
- Sponsor's protocol ID
- VWD-001
- Status last verified
- Apr 2026
- First submitted
- 21 Jun 2022
- Last updated
- 22 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Severe Von Willebrand Disease Patient Registry: A Longitudinal Natural History and Patient Outcomes Study
- ClinicalTrials.gov ID
- NCT05437536
- Acronym / sponsor ID
- VWD-001
- Conditions
- VWD - Von Willebrand's Disease
- Keywords
- 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
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- VWD Connect FoundationUniversity, hospital or other
- Study start
- 10 Dec 2021 (actual)
- Primary completion
- Dec 2031 (estimated)
- Study completion
- Dec 2031 (estimated)
- First posted
- 29 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Case only
- Time perspective
- Other
- Target follow-up
- 5 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals diagnosed with severe VWD
Key inclusion criteria (7 listed)
- Anyone meeting the diagnostic classification for von Willebrand disease
- Any VWF antigen or activity:
- Provide informed consent by participant or legally authorized representative
- Be willing and able to comply with study procedures and data collection
- Reside in the United States
Key exclusion criteria (1 listed)
- 1. Have a clinical diagnosis of acquired VWD
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Outcomes are not applicable; this study is a patient registry.
Time frame: At least 5 years
Statistical analyses will focus on simple characterization of the registry.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2026.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Christopher Walsh, MD, PhD principal investigator
Mt. Sinai School of Medicine
Mrinal Gounder, MD principal investigator
Memorial Sloan Kettering Cancer Center
Christina Morgenthaler, MS, MBA principal investigator
VWD Connect Foundation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of VWD - Von Willebrand's Disease, run by VWD Connect Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Christina Morgenthaler, MS, MBA ((279) 346-6202, morgenthaler@vwdregistry.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Outcomes are not applicable; this study is a patient registry. (time frame: At least 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2026.