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

Purpose

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.

Condition

  • Von Willebrand Disease, Type 3

Eligibility

Eligible Ages
Over 2 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
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.
  • Drug: Von Willebrand Factor Concentrates
    Used according to local labeling or local treatment guidelines.
  • Drug: Von Willebrand Factor Concentrates and Factor VIII Concentrates
    Used according to local labeling or local treatment guidelines.
  • Drug: Factor VIII Concentrates
    Used according to local labeling or local treatment guidelines.
  • Drug: Recombinant Activated Factor VII
    Used according to local labeling or local treatment guidelines.
  • Drug: Activated Prothrombin Complex Concentrate
    Used according to local labeling or local treatment guidelines.

Recruiting Locations

UC Davis
Sacramento, California 95817

University of Florida
Gainesville, Florida 32610

University of Minnesota Medical Center
Minneapolis, Minnesota 55455

Washington University School of Medicine
St Louis, Missouri 63110

More Details

Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: WP45335 https://forpatients.roche.com/
888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com