Clinical trial NCT05437536: The Severe Von Willebrand Disease (sVWD) Patient Registry
Official title: The Severe Von Willebrand Disease Patient Registry: a Longitudinal Natural History and Patient Outcomes Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
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
Lead sponsor: VWD Connect Foundation
Sponsor class: OTHER
First posted: 2022-06-29
Enrollment: 400
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Individuals diagnosed with severe VWD

Brief 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. 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 and regular intervals. 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 a questionnaire in the registry website within a 12-month period will be contacted by registry personnel for follow-up.

Eligibility criteria: Inclusion Criteria:

* Have a clinical diagnosis of Von Willebrand Disease (VWD) with any Von Willebrand factor level(s) \< 20%
* 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 criterion 1, 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 Von Willebrand factor levels \> 20% 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:

* Have a clinical diagnosis of acquired VWD

Primary outcomes:
measure: Outcomes are not applicable; this study is a patient registry.
timeFrame: At least 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Wellington
country: United States
facility: VWD Connect Foundation
state: Florida
Study officials:
affiliation: Mt. Sinai School of Medicine
name: Christopher Walsh, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Memorial Sloan Kettering Cancer Center
name: Mrinal Gounder, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: VWD Connect Foundation
name: Christina Morgenthaler, MS, MBA
role: PRINCIPAL_INVESTIGATOR
References: