Von Willebrand DiseasesNCT03853486
ATHN 9: Severe VWD Natural History Study
An observational study of Von Willebrand Diseases, run by American Thrombosis and Hemostasis Network at 22 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Von Willebrand Diseases
- How many people
- 108 participants
- Where
- 22 sitesUnited States
- Study dates
- 18 Jun 2019to 26 Jun 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
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
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- 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).
Trial locations and contacts
22 sites in 1 country.
22 sites in 1 country
United States22 sites
University of Michigan Hemophilia and Coagulation Disorders
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta/Emory
Atlanta, Georgia, 30322
University of Colorado Denver Hemophilia and Thrombosis Center
Aurora, Colorado, 80045
Nationwide Children's Hospital Columbus
Columbus, Ohio, 43205
Children's Hospital of Michigan Hemostasis and Thrombosis Center
Detroit, Michigan, 48201
Michigan State University Center for Bleeding and Clotting Disorders
East Lansing, Michigan, 48823
Connecticut Bleeding and Clotting Disorders Center
Farmington, Connecticut, 06030
University of Florida Hemophilia Treatment Center
Gainesville, Florida, 32610
Hemophilia Outreach Center
Green Bay, Wisconsin, 54311
Indiana Hemophilia and Thrombosis Center (IHTC)
Indianapolis, Indiana, 46260
University of Tennessee, University Clinical Health (Memphis)
Memphis, Tennessee, 38119
Versiti - Blood Center of Wisconsin
Milwaukee, Wisconsin, 53201
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, 70112
Center for Inherited Blood Disorders
Orange, California, 92868
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61615
Pennsylvania Comprehensive Hemophilia and Thrombophilia Program / Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
Oregon Health
Portland, Oregon, 91239
Rhode Island Hemostasis & Thrombosis Center
Providence, Rhode Island, 02903
Mayo Comprehensive Hemophilia Center
Rochester, Minnesota, 55905
Washington University Center for Treatment of Bleeding and Blood Clotting Disorders
St Louis, Missouri, 63110
About this study
Official title: ATHN 9: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Severe Von Willebrand Disease (VWD)
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.
Read the detailed description
The overarching objective of this longitudinal, observational and prospective study is to characterize the safety and effectiveness of factor replacement in participants with clinically severe congenital VWD (VWF:Ag, VWF:GPlbM or VWF:RCo of ≤30% or ≤40% of normal with severe bleeding phenotype defined as requiring recurrent use of factor concentrates) enrolled in the ATHNdataset.
This is a longitudinal, observational cohort study being conducted at up to 30 ATHN-affiliated sites. Participants will be followed for 2 years from time of study enrolment. The total study duration is 3 years.
Safety will be measured by the number of reported events defined by the European Haemophilia Safety Surveillance (EUHASS) program. In addition, although not specifically defined by EUHASS, treatment-emergent side effects of therapy will be included as reportable events including: hypersensitivity/allergic reactions, thrombotic events, VW Factor inhibitor development, treatment-emergent side effects of therapy, transfusion-transmitted infections, malignancy, cardiovascular events, neurological events, unexpected poor efficacy and death.
Secondary objectives of ATHN 9 are:
- to enrich and analyze the data from currently enrolled participants with clinically severe congenital VWD in the ATHNdataset via the collection of laboratory data consisting of a standardized diagnostic battery using an ELISA based VWF activity assay, and genetic sequence analysis of VWF coding regions and adjacent non-coding regions;
- to establish a platform for sub-studies for participants with congenital severe VWD, that are treated with VWF products on demand or have started on or switched to a particular VWF containing product for prophylaxis;
- to evaluate the use of factor replacement as prophylaxis in participants over 6-month time periods;
- to describe bleeding events, changes in overall bleeding and annualized bleeding rate (ABR) over the course of the study as measured by individual bleeding components; and
- to describe real-world effectiveness of VWD treatment as measured by health care utilization and quality of life.
- Conditions
- Von Willebrand Diseases
- Keywords
- VWF, von Willebrand Disease, VWD, Factor, ATHN 9
- Medical subject headings
- von Willebrand Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
To enrich and analyze the data from currently enrolled participants with clinically severe congenital VWD in the ATHNdataset via the collection of laboratory data consisting of a standardized diagnostic battery using an ELISA-based VWF assay.
Measured: 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.
To enrich and analyze the data from currently enrolled participants with clinically-severe congenital VWD in the ATHNdataset via the collection of laboratory data using genetic sequence analysis of VWF coding regions and adjacent non-coding regions.
Measured: 2 years
Substudy modules will be developed to evaluate and report on cohorts of study participants who initiate treatment with specific product.
To measure the number of participants taking unique VWF products.
Measured: 2 years
Factor replacement used as prophylaxis.
Report number of particpants using factor replacement as prophylaxis.
Measured: 3 years
Capture bleeding events using the Pictorial Bleeding. Assessment Chart.
The number of participants with bleeding events analyzed over the course of the study.
Measured: 3 years
Capture annualized bleeding rate (ABR) using ISTH BAT Assessment Tool.
The change in the annualized bleeding rate (ABR) for participants over the course of the study by analyzing the number of bleeding events divided by the length of time of the treatment (in years).
Measured: 3 years
Calculate the effectiveness of VWD treatment as measured by health care utilization.
The number of visits/hospitalizations.
Measured: 3 years
Analyze the effectivness of VWD treatment as measured by score on PROMIS questionnaire using the 7 PROMIS domains (depression; anxiety; physical function; pain; fatigue; sleep disturbance; and participation in social roles and activities).
Health-related Quality of Life measured annually by the Patient Reported Outcomes Measurement Information System (PROMIS ®) Profile.
Measured: 3 years
Capture bleeding events using the Pictorial Bleeding Assessment Chart.
The number of participants with bleeding events analyzed over the course of the study.
Measured: 3 years
Capture annualized bleeding rates (ABR) using the Pictorial Bleeding Assessment Chart.
The change in the annualized bleeding rate (ABR) for participants over the course of the study by analyzing the number of bleeding events divided by the length of time of the treatment (in years).
Measured: 3 years
Calculate the success of VWD treatment as measured by health care utilization.
The types of visits/hospitalizations
Measured: 3 years
Capture the effectiveness of VWD treatments using health-related quality of life.
Measure walking ability as part of quality of life using the V-WIQ questionnaire.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2019
- Study start18 Jun 2019
- Primary completion12 Jun 2026
- Study completion26 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand DiseasesStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 108 participants (actual)
Sponsor and investigators
- Lead sponsor
- American Thrombosis and Hemostasis Network
- Sponsor type
- Research network
- Collaborators
- Takeda
- Responsible party
- Sponsor
- Investigators
- Robert Sidonio, MD, Principal investigator, Emory University / Children's Healthcare of AtlantaAngela Weyand, MD, Principal investigator, University of Michigan Hemophilia and Coagulation Disorders
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03853486
- Sponsor's protocol ID
- ATHN 9
- Status last verified
- Apr 2026
- First submitted
- 11 Jan 2019
- Last updated
- 13 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ATHN 9: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Severe Von Willebrand Disease (VWD)
- ClinicalTrials.gov ID
- NCT03853486
- Acronym / sponsor ID
- ATHN 9
- Conditions
- Von Willebrand Diseases
- Keywords
- VWF, von Willebrand Disease, VWD, Factor, ATHN 9
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 108 participants (actual)
- Lead sponsor
- American Thrombosis and Hemostasis NetworkResearch network
- Collaborators
- Takeda
- Study start
- 18 Jun 2019 (actual)
- Primary completion
- 12 Jun 2026 (actual)
- Study completion
- 26 Jun 2026 (actual)
- First posted
- 25 Feb 2019 (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.
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.
Detailed description
The overarching objective of this longitudinal, observational and prospective study is to characterize the safety and effectiveness of factor replacement in participants with clinically severe congenital VWD (VWF:Ag, VWF:GPlbM or VWF:RCo of ≤30% or ≤40% of normal with severe bleeding phenotype defined as requiring recurrent use of factor concentrates) enrolled in the ATHNdataset.
This is a longitudinal, observational cohort study being conducted at up to 30 ATHN-affiliated sites. Participants will be followed for 2 years from time of study enrolment. The total study duration is 3 years.
Safety will be measured by the number of reported events defined by the European Haemophilia Safety Surveillance (EUHASS) program. In addition, although not specifically defined by EUHASS, treatment-emergent side effects of therapy will be included as reportable events including: hypersensitivity/allergic reactions, thrombotic events, VW Factor inhibitor development, treatment-emergent side effects of therapy, transfusion-transmitted infections, malignancy, cardiovascular events, neurological events, unexpected poor efficacy and death.
Secondary objectives of ATHN 9 are:
- to enrich and analyze the data from currently enrolled participants with clinically severe congenital VWD in the ATHNdataset via the collection of laboratory data consisting of a standardized diagnostic battery using an ELISA based VWF activity assay, and genetic sequence analysis of VWF coding regions and adjacent non-coding regions;
- to establish a platform for sub-studies for participants with congenital severe VWD, that are treated with VWF products on demand or have started on or switched to a particular VWF containing product for prophylaxis;
- to evaluate the use of factor replacement as prophylaxis in participants over 6-month time periods;
- to describe bleeding events, changes in overall bleeding and annualized bleeding rate (ABR) over the course of the study as measured by individual bleeding components; and
- to describe real-world effectiveness of VWD treatment as measured by health care utilization and quality of life.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (2 listed)
- 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).
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
Reported adverse events from VWF regimens for different indications (on-demand, surgery, and prophylaxis) as measured by EUHASS.
Time frame: 2 years
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.
12 secondary outcomes
Enrich and analyze data collected about AE events as defined by EUHASS using standardized diagnostic battery using an ELISA-based VWF assay.
Time frame: 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.
Time frame: 2 years
Substudy modules will be developed to evaluate and report on cohorts of study participants who initiate treatment with specific product.
Time frame: 2 years
Factor replacement used as prophylaxis.
Time frame: 3 years
Capture bleeding events using the Pictorial Bleeding. Assessment Chart.
Time frame: 3 years
Capture annualized bleeding rate (ABR) using ISTH BAT Assessment Tool.
Time frame: 3 years
Calculate the effectiveness of VWD treatment as measured by health care utilization.
Time frame: 3 years
Analyze the effectivness of VWD treatment as measured by score on PROMIS questionnaire using the 7 PROMIS domains (depression; anxiety; physical function; pain; fatigue; sleep disturbance; and participation in social roles and activities).
Time frame: 3 years
Capture bleeding events using the Pictorial Bleeding Assessment Chart.
Time frame: 3 years
Capture annualized bleeding rates (ABR) using the Pictorial Bleeding Assessment Chart.
Time frame: 3 years
Calculate the success of VWD treatment as measured by health care utilization.
Time frame: 3 years
Capture the effectiveness of VWD treatments using health-related quality of life.
Time frame: 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jul 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Robert Sidonio, MD principal investigator
Emory University / Children's Healthcare of Atlanta
Angela Weyand, MD principal investigator
University of Michigan Hemophilia and Coagulation Disorders
22 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Diseases, run by American Thrombosis and Hemostasis Network at 22 sites 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 3 inclusion and 2 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: finished, as of the sponsor's last update on 13 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reported adverse events from VWF regimens for different indications (on-demand, surgery, and prophylaxis) as measured by EUHASS. (time frame: 2 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 13 Jul 2026.