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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03853486
Other study ID # ATHN 9
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date March 18, 2019
Est. completion date January 31, 2025

Study information

Verified date January 2024
Source American Thrombosis and Hemostasis Network
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical 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.


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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 108
Est. completion date January 31, 2025
Est. primary completion date December 31, 2024
Accepts healthy volunteers
Gender All
Age group N/A and older
Eligibility 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).

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
United States University of Michigan Hemophilia and Coagulation Disorders Ann Arbor Michigan
United States Children's Healthcare of Atlanta/Emory Atlanta Georgia
United States University of Colorado Denver Hemophilia and Thrombosis Center Aurora Colorado
United States Nationwide Children's Hospital Columbus Columbus Ohio
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 Connecticut Bleeding and Clotting Disorders Center Farmington Connecticut
United States University of Florida Hemophilia Treatment Center Gainesville Florida
United States Hemophilia Outreach Center Green Bay Wisconsin
United States Indiana Hemophilia and Thrombosis Center (IHTC) Indianapolis Indiana
United States University of Tennessee, University Clinical Health (Memphis) Memphis Tennessee
United States Versiti - Blood Center of Wisconsin Milwaukee Wisconsin
United States Vanderbilt University Medical Center Nashville Tennessee
United States Louisiana Center for Bleeding and Clotting Disorders New Orleans Louisiana
United States Center for Inherited Blood Disorders Orange California
United States Bleeding and Clotting Disorders Institute Peoria Illinois
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 Oregon Health Portland Oregon
United States Rhode Island Hemostasis & Thrombosis Center Providence Rhode Island
United States Mayo Comprehensive Hemophilia Center Rochester Minnesota
United States Washington University Center for Treatment of Bleeding and Blood Clotting Disorders Saint Louis Missouri

Sponsors (2)

Lead Sponsor Collaborator
American Thrombosis and Hemostasis Network Takeda

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 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. 3 years
Secondary 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. 2 years
Secondary 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. 2 years
Secondary Factor replacement used as prophylaxis. Report number of particpants using factor replacement as prophylaxis. 3 years
Secondary Capture bleeding events using the Pictorial Bleeding. Assessment Chart. The number of participants with bleeding events analyzed over the course of the study. 3 years
Secondary 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). 3 years
Secondary Calculate the effectiveness of VWD treatment as measured by health care utilization. The number of visits/hospitalizations. 3 years
Secondary 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. 3 years
Secondary Capture bleeding events using the Pictorial Bleeding Assessment Chart. The number of participants with bleeding events analyzed over the course of the study. 3 years
Secondary 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). 3 years
Secondary Calculate the success of VWD treatment as measured by health care utilization. The types of visits/hospitalizations 3 years
Secondary 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. 3 years
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