ATHN 9: Severe Von Willebrand Disease Natural History Study
Overview
Tab Title Description
Study type
ObservationalDescribes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
Study IDs
Site IRB
- Rochester, Minnesota: 21-013017
About this study
The purpose of this study is to assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.
Participation eligibility
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.
Inclusion Criteria:
- Males and females.
- Participants with severe Von Willebrand Disease with Type 3 VWD or VWF:RCo, VWF:GPIbM or VWF:Ag ≤ 30% of pooled normal control plasma on more than one occasion.
- Participants with clinically severe VWD as defined by VWF:RCo or VWF:Ag ≤ 40% of normal with severe bleeding phenotype defined as requiring use of recurrent factor concentrates.
- Co-enrollment in the ATHN dataset.
Exclusion Criteria
- Diagnosis of platelet-type VWD;
- Diagnosis of acquired VWD (clinical diagnosis made by the hemophilia health care provider, typically based on association with hypothyroidism, lymphoproliferative and myeloproliferative disorders, malignancies and cardiovascular disease, typically aortic stenosis or LVAD).
Eligibility last updated 12/15/21. Questions regarding updates should be directed to the study team contact.
Participating Mayo Clinic locations
Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.
| Mayo Clinic Location |
Status |
|
Rochester, Minn.
Mayo Clinic principal investigator Rajiv Pruthi, M.B.B.S. |
Closed for enrollment |
|
More information
Publications
Publications are currently not available