A Multicenter Non-Interventional Study Evaluating Bleeds and Health-Related Quality Of Life in Patients With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Status: Recruiting
Location: See all (23) locations...
Intervention Type: Drug
Study Type: Observational
SUMMARY
This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:
• Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
• Adequate hematologic, hepatic, and renal function
• Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study
• For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements
Locations
United States
California
UC Davis
RECRUITING
Sacramento
Missouri
Washington University School of Medicine
RECRUITING
St Louis
Other Locations
Belgium
UZ Leuven Gasthuisberg
RECRUITING
Leuven
Canada
McGill University Health Center
RECRUITING
Montreal
The Hospital for Sick Children
RECRUITING
Toronto
Colombia
IPS SURA Industriales Medellín
RECRUITING
Medellín
France
Hopital Claude Huriez - CHU Lille
RECRUITING
Lille
Groupe Hospitalier Necker Enfants Malades
RECRUITING
Paris
Germany
Universitätsklinikum Bonn
RECRUITING
Bonn
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
RECRUITING
Duisburg
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
RECRUITING
Frankfurt/m.
Italy
AOU Careggi
RECRUITING
Florence
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
RECRUITING
Rome
Japan
Kurume University Hospital
RECRUITING
Fukuoka
Tokyo Medical University Hospital
RECRUITING
Tokyo
Netherlands
Erasmus MC
RECRUITING
Rotterdam
Poland
Instytut Hematologii i Transfuzjologii
RECRUITING
Warsaw
Spain
Hospital Universitario la Paz
RECRUITING
Madrid
Hospital Universtiario Virgen del Rocio
RECRUITING
Seville
Sweden
Sahlgrenska Universitetssjukhuset
RECRUITING
Gothenburg
United Kingdom
Great Ormond Street Hospital
RECRUITING
London
St Thomas' Hospital
RECRUITING
London
Manchester Royal Infirmary
RECRUITING
Manchester
Contact Information
Primary
Reference Study ID Number: WP45335 https://forpatients.roche.com/
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. Only)
Time Frame
Start Date:2025-04-29
Estimated Completion Date:2026-11-01
Participants
Target number of participants:40
Treatments
Participants with Type 3 VWD Treated with Prophylactic SOC
Participants with Type 3 VWD, aged 2 years and above, who are currently on standard of care (SOC) prophylactic therapy are anticipated to remain on the chosen SOC regimen during the study. Dosing and treatment duration of any studied medicinal products collected as part of this study are at the discretion of the treating physician in accordance with local labeling or local treatment guidelines.