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

Last updated: June 25, 2025
Sponsor: Hoffmann-La Roche
Overall Status: Active - Recruiting

Phase

N/A

Condition

Von Willebrand Disease

Treatment

Activated Prothrombin Complex Concentrate

Factor VIII Concentrates

Von Willebrand Factor Concentrates

Clinical Study ID

NCT06883240
WP45335
  • Ages > 2
  • All Genders

Study 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 Criteria

Inclusion

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 timesweekly, as per prescribed dose) and anticipation to remain on the same regimenduring the study

  • For participants of childbearing potential: agreement to remain abstinent or adhereto the contraception requirements

Exclusion

Exclusion Criteria:

  • Inherited or acquired bleeding disorder other than Congenital Type 3 VWD

  • History of gastrointestinal bleeding within 18 months prior to enrollment, or anyprevious diagnosis of angiodysplasia

  • History of intracranial hemorrhage

  • Previous or current treatment for thromboembolic disease or signs of thromboembolicdisease

  • Other conditions (e.g., certain autoimmune diseases) that may increase risk ofbleeding or thrombosis

  • History of clinically significant hypersensitivity associated with monoclonalantibody therapies or components of the emicizumab injection

  • Use of systemic immunomodulators (e.g., interferon) at enrollment or planned useduring the study, with the exception of anti-retroviral therapy

Study Design

Total Participants: 40
Treatment Group(s): 5
Primary Treatment: Activated Prothrombin Complex Concentrate
Phase:
Study Start date:
April 29, 2025
Estimated Completion Date:
November 01, 2026

Connect with a study center

  • UZ Leuven Gasthuisberg

    Leuven, 3000
    Belgium

    Active - Recruiting

  • The Hospital for Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Active - Recruiting

  • IPS SURA Industriales Medellín

    Medellin,
    Colombia

    Active - Recruiting

  • Hopital Claude Huriez - CHU Lille

    Lille cedex, 59037
    France

    Active - Recruiting

  • Universitätsklinikum Bonn

    Bonn, 53127
    Germany

    Active - Recruiting

  • Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

    Duisburg, 47051
    Germany

    Active - Recruiting

  • Universita' Degli Studi La Sapienza-Ist.Di Ematologia

    Roma, Lazio 00161
    Italy

    Active - Recruiting

  • AOU Careggi

    Firenze, Toscana 50134
    Italy

    Active - Recruiting

  • Kurume University Hospital

    Fukuoka, 830-0011
    Japan

    Active - Recruiting

  • Hospital Universitario la Paz

    Madrid, 28046
    Spain

    Active - Recruiting

  • Hospital Universitario Virgen del Rocio

    Sevilla, 41013
    Spain

    Active - Recruiting

  • Great Ormond Street Hospital

    London, WC1N 3JH
    United Kingdom

    Active - Recruiting

  • Manchester Royal Infirmary

    Manchester, M13 9WL
    United Kingdom

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.