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Набира участници NCT06883240

An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

Заболявания: Von Willebrand Disease, Type 3

Спонсор: Hoffmann-La Roche

Налично на: БГ
Обобщение
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.
Кой може да участва
Inclusion Criteria: * 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 Exclusion Criteria: * Inherited or acquired bleeding disorder other than Congenital Type 3 VWD * History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia * History of intracranial hemorrhage * Previous or current treatment for thromboembolic disease or signs of thromboembolic disease * Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
Интервенции
Von Willebrand Factor Concentrates
DRUG
Von Willebrand Factor Concentrates and Factor VIII Concentrates
DRUG
Factor VIII Concentrates
DRUG
Recombinant Activated Factor VII
DRUG
Activated Prothrombin Complex Concentrate
DRUG
Места на провеждане 27
Белгия (1)
UZ Leuven Gasthuisberg
Leuven
Канада (2)
McGill University Health Center
Montreal , Quebec
The Hospital for Sick Children
Toronto , Ontario
COMPLETED
Colombia (1)
IPS SURA Industriales Medellín
Medellín
Франция (2)
Hopital Claude Huriez - CHU Lille
Lille
Groupe Hospitalier Necker Enfants Malades
Paris
Германия (3)
Universitätsklinikum Bonn
Bonn
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M.
Италия (3)
AOU Careggi
Florence , Tuscany
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan , Lombardy
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome , Lazio
Япония (2)
Kurume University Hospital
Fukuoka
COMPLETED
Tokyo Medical University Hospital
Tokyo
Нидерландия (1)
Erasmus MC
Rotterdam
Полша (1)
Instytut Hematologii i Transfuzjologii
Warsaw
South Africa (1)
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg
Испания (2)
Hospital Universitario la Paz
Madrid
Hospital Universtiario Virgen del Rocio
Seville
Швеция (1)
Sahlgrenska Universitetssjukhuset
Gothenburg
Великобритания (3)
St Thomas' Hospital
London
Great Ormond Street Hospital
London
Manchester Royal Infirmary
Manchester
САЩ (4)
University of Florida
Gainesville , Florida
University of Minnesota Medical Center
Minneapolis , Minnesota
UC Davis
Sacramento , California
Washington University School of Medicine
St Louis , Missouri
Технически детайли
Статус
Набира участници
Вид изследване
OBSERVATIONAL
Пол
Мъже и жени
Минимална възраст
2 Years
Здрави доброволци
Не
Начална дата
29.04.2025
Крайна дата
01.11.2026
Регистрационен номер
NCT06883240
Източник
clinicaltrials.gov
Запитване за медицински туризъм

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