ATHN 9: Severe VWD Natural History Study
ATHN 9: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Severe Von Willebrand Disease (VWD)
1 other identifier
observational
108
1 country
22
Brief Summary
ATHN 9 is a natural history study 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Jun 2019
Longer than P75 for all trials
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 11, 2019
CompletedFirst Posted
Study publicly available on registry
February 25, 2019
CompletedStudy Start
First participant enrolled
June 18, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 31, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
July 30, 2026
ExpectedMarch 12, 2025
March 1, 2025
6.5 years
January 11, 2019
March 11, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Reported adverse events from VWF regimens for different indications (on-demand, surgery, and prophylaxis) as measured by EUHASS.
Number of adverse events as measured by EUHASS as well as treatment-emergent side effects of therapy for various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.
2 years
Secondary Outcomes (12)
Enrich and analyze data collected about AE events as defined by EUHASS using standardized diagnostic battery using an ELISA-based VWF assay.
3 years
Enrich and analyze data collected about AE events, as defined by EUHASS using genetic sequence analysis of VWF coding regions and adjacent non-coding regions.
2 years
Substudy modules will be developed to evaluate and report on cohorts of study participants who initiate treatment with specific product.
2 years
Factor replacement used as prophylaxis.
3 years
Capture bleeding events using the Pictorial Bleeding. Assessment Chart.
3 years
- +7 more secondary outcomes
Eligibility Criteria
This is a natural history study in which it is anticipated that approximately 130 participants will be enrolled. The study will attempt to enroll a target number of at least 30 participants who are receiving VONVENDI but will not mandate the use of VONVENDI.
You may qualify if:
- Participants with severe Von Willebrand Disease with Type 3 VWD or VWF:RCo, VWF:GPlbM or VWF:Ag ≤30% of pooled normal control plasma on more than one occasion;
- Participants with clinically severe VWD as defined by VWF:RCo, VWF:GPlbM or VWF:Ag ≤40% of normal with severe bleeding phenotype defined as requiring recurrent use of factor concentrates; and
- Co-enrollment in the ATHNdataset.
You may not qualify if:
- Diagnosis of platelet-type VWD;
- Diagnosis of acquired VWD (clinical diagnosis based on association with hypothyroidism, lymphoproliferative and myeloproliferative disorders, malignancies and cardiovascular disease, typically aortic stenosis or LVAD).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- American Thrombosis and Hemostasis Networklead
- Takedacollaborator
Study Sites (22)
Center for Inherited Blood Disorders
Orange, California, 92868, United States
University of Colorado Denver Hemophilia and Thrombosis Center
Aurora, Colorado, 80045, United States
Connecticut Bleeding and Clotting Disorders Center
Farmington, Connecticut, 06030, United States
University of Florida Hemophilia Treatment Center
Gainesville, Florida, 32610, United States
Children's Healthcare of Atlanta/Emory
Atlanta, Georgia, 30322, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61615, United States
Indiana Hemophilia and Thrombosis Center (IHTC)
Indianapolis, Indiana, 46260, United States
Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, 70112, United States
University of Michigan Hemophilia and Coagulation Disorders
Ann Arbor, Michigan, 48109, United States
Children's Hospital of Michigan Hemostasis and Thrombosis Center
Detroit, Michigan, 48201, United States
Michigan State University Center for Bleeding and Clotting Disorders
East Lansing, Michigan, 48823, United States
Mayo Comprehensive Hemophilia Center
Rochester, Minnesota, 55905, United States
Washington University Center for Treatment of Bleeding and Blood Clotting Disorders
St Louis, Missouri, 63110, United States
Nationwide Children's Hospital Columbus
Columbus, Ohio, 43205, United States
Oregon Health
Portland, Oregon, 91239, United States
Pennsylvania Comprehensive Hemophilia and Thrombophilia Program / Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213, United States
Rhode Island Hemostasis & Thrombosis Center
Providence, Rhode Island, 02903, United States
University of Tennessee, University Clinical Health (Memphis)
Memphis, Tennessee, 38119, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
Hemophilia Outreach Center
Green Bay, Wisconsin, 54311, United States
Versiti - Blood Center of Wisconsin
Milwaukee, Wisconsin, 53201, United States
Biospecimen
All participants will undergo laboratory evaluation. The remaining specimen volume will be stored and may be used for future research. Specimens will be tested as follows: * FVIII Activity, VWF Antigen, and the VWF:GPIbM Activity Assay. Reflex testing may include: VWD Type 2B Binding, VWD Type 2N Binding, VWF Propeptide Antigen, and/or VWF Quantitative Multimers * Genetic sequence analysis of VWF coding regions and adjacent non-coding regions
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Robert Sidonio, MD
Emory University / Children's Healthcare of Atlanta
- PRINCIPAL INVESTIGATOR
Angela Weyand, MD
University of Michigan Hemophilia and Coagulation Disorders
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 11, 2019
First Posted
February 25, 2019
Study Start
June 18, 2019
Primary Completion
December 31, 2025
Study Completion (Estimated)
July 30, 2026
Last Updated
March 12, 2025
Record last verified: 2025-03
Data Sharing
- IPD Sharing
- Will not share