Ignite Creation Date:
2024-05-06 @ 12:48 PM
Last Modification Date:
2024-10-26 @ 1:04 PM
Study NCT ID:
NCT03853486
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-01-30
First Post:
2019-01-11
Brief Title:
ATHN 9 Severe VWD Natural History Study
Sponsor:
American Thrombosis and Hemostasis Network
Organization:
American Thrombosis and Hemostasis Network
Study Overview
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
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 VWFAg VWFGPlbM or VWFRCo 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 hypersensitivityallergic 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
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 hypersensitivityallergic 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None