A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Severe Von Willebrand Disease (VWD) (ATHN 9: Severe VWD Natural History Study)

Study identification

• EU PAS number:
  EUPAS34614
• Study ID:
  34615
• Official title and acronym:
  A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Severe Von Willebrand Disease (VWD) (ATHN 9: Severe VWD Natural History Study)
• DARWIN EU® study:
  No
• Study countries:
  United States
• Study 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 American Thrombosis and Hemostasis Network (ATHN) ATHNdataset. This is a longitudinal, observational cohort study being conducted at up to at least 30 ATHN-affiliated sites. Participants will be followed for 2 years from time of study enrollment. The total study duration is 3 years. The primary objective is to assess the safety of various VWF treatment regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD. Safety will be measured by the number of reported events defined by the European Haemophilia Safety Surveillance (EUHASS) program. All treatment regimens will be at the discretion of the participant’s hemophilia healthcare providers. Investigators will emphasize that clinical care and participation in the study is not determined based on their selection of clotting factor replacement or non-factor products. No treatment will be provided by the study. All study visits, procedures and follow-up will be timed to coincide with routine, scheduled bleeding disorder care whenever possible.
• Study status:
  Ongoing

Research institutions and networks

Institutions

American Thrombosis and Hemostasis Network (ATHN)

Study timelines

Date when funding contract was signed

Actual: 14/12/2017

Study start date

Actual: 04/09/2019

Data analysis start date

Planned: 29/08/2020

Date of interim report, if expected

Planned: 29/12/2020

Date of final study report

Planned: 20/06/2023

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Takeda

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable

Other study registration identification numbers and links

ClinicalTrials.gov Identifier: NCT03853486