ATHN 9: Severe VWD Natural History Study

NCT ID: NCT03853486

Last Updated: 2025-03-12

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 108 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-06-18
• Study Completion Date: 2026-07-30

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.

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

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

1. 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;

2. 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

3. Co-enrollment in the ATHNdataset.

Exclusion Criteria

1. Diagnosis of platelet-type VWD;

2. Diagnosis of acquired VWD (clinical diagnosis based on association with hypothyroidism, lymphoproliferative and myeloproliferative disorders, malignancies and cardiovascular disease, typically aortic stenosis or LVAD).

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Takeda

INDUSTRY

Sponsor Role: collaborator

American Thrombosis and Hemostasis Network

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Robert Sidonio, MD

Role: PRINCIPAL_INVESTIGATOR

Emory University / Children's Healthcare of Atlanta

Angela Weyand, MD

Role: PRINCIPAL_INVESTIGATOR

University of Michigan Hemophilia and Coagulation Disorders

Locations

Center for Inherited Blood Disorders

Orange, California, United States

University of Colorado Denver Hemophilia and Thrombosis Center

Aurora, Colorado, United States

Connecticut Bleeding and Clotting Disorders Center

Farmington, Connecticut, United States

University of Florida Hemophilia Treatment Center

Gainesville, Florida, United States

Children's Healthcare of Atlanta/Emory

Atlanta, Georgia, United States

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States

Indiana Hemophilia and Thrombosis Center (IHTC)

Indianapolis, Indiana, United States

Louisiana Center for Bleeding and Clotting Disorders

New Orleans, Louisiana, United States

University of Michigan Hemophilia and Coagulation Disorders

Ann Arbor, Michigan, United States

Children's Hospital of Michigan Hemostasis and Thrombosis Center

Detroit, Michigan, United States

Michigan State University Center for Bleeding and Clotting Disorders

East Lansing, Michigan, United States

Mayo Comprehensive Hemophilia Center

Rochester, Minnesota, United States

Washington University Center for Treatment of Bleeding and Blood Clotting Disorders

St Louis, Missouri, United States

Nationwide Children's Hospital Columbus

Columbus, Ohio, United States

Oregon Health

Portland, Oregon, United States

Pennsylvania Comprehensive Hemophilia and Thrombophilia Program / Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Rhode Island Hemostasis & Thrombosis Center

Providence, Rhode Island, United States

University of Tennessee, University Clinical Health (Memphis)

Memphis, Tennessee, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Hemophilia Outreach Center

Green Bay, Wisconsin, United States

Versiti - Blood Center of Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

Other Identifiers

ATHN 9

Identifier Type: -

Identifier Source: org_study_id