Observational study on treatment for Type 3 Von Willebrand Disease
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
This study is looking at how well standard treatments work for people with Type 3 von Willebrand disease and how these treatments affect their quality of life over at least 24 weeks.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 40 (estimated) |
| Ages | 2 Years and up |
| Sex | All |
| Sponsor | Hoffmann-La Roche Industry-sponsored |
| Drugs / interventions | emicizumab |
| Locations | 27 sites (Sacramento, California and 26 other locations) |
| Trial ID | NCT06883240 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather data on the effectiveness and safety of standard-of-care prophylactic treatments for participants diagnosed with Type 3 von Willebrand disease. Over a minimum observation period of 24 weeks, the study will assess health-related quality of life and monitor treatment outcomes in routine clinical settings. Participants will receive various treatments, including Von Willebrand Factor Concentrates and Factor VIII Concentrates, as per their standard care regimen.
Who should consider this trial
Good fit: Ideal candidates include individuals with a confirmed diagnosis of Type 3 von Willebrand disease who are currently receiving prophylactic therapy.
Not a fit: Patients with other bleeding disorders or significant medical histories that complicate treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of treatment effectiveness and improve quality of life for patients with Type 3 von Willebrand disease.
How similar studies have performed: Other observational studies have shown success in assessing treatment outcomes for bleeding disorders, indicating that this approach is supported by prior research.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Sacramento, California and 26 other locations
- UC Davis — Sacramento, California, United States (Recruiting)
- University of Florida — Gainesville, Florida, United States (Recruiting)
- University of Minnesota Medical Center — Minneapolis, Minnesota, United States (Recruiting)
- Washington University School of Medicine — St Louis, Missouri, United States (Recruiting)
- UZ Leuven Gasthuisberg — Leuven, Belgium (Recruiting)
- The Hospital for Sick Children — Toronto, Ontario, Canada (Completed)
- McGill University Health Center — Montreal, Quebec, Canada (Recruiting)
- IPS SURA Industriales Medellín — Medellín, Colombia (Recruiting)
- Hopital Claude Huriez - CHU Lille — Lille, France (Recruiting)
- Groupe Hospitalier Necker Enfants Malades — Paris, France (Recruiting)
- Universitätsklinikum Bonn — Bonn, Germany (Recruiting)
- Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz — Duisburg, Germany (Recruiting)
- Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin — Frankfurt/M., Germany (Recruiting)
- Universita' Degli Studi La Sapienza-Ist.Di Ematologia — Rome, Lazio, Italy (Recruiting)
- IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan, Lombardy, Italy (Recruiting)
- AOU Careggi — Florence, Tuscany, Italy (Recruiting)
- Kurume University Hospital — Fukuoka, Japan (Completed)
- Tokyo Medical University Hospital — Tokyo, Japan (Recruiting)
- Erasmus MC — Rotterdam, Netherlands (Recruiting)
- Instytut Hematologii i Transfuzjologii — Warsaw, Poland (Recruiting)
- Charlotte Maxeke Johannesburg Academic Hospital — Johannesburg, South Africa (Recruiting)
- Hospital Universitario la Paz — Madrid, Spain (Recruiting)
- Hospital Universtiario Virgen del Rocio — Seville, Spain (Recruiting)
- Sahlgrenska Universitetssjukhuset — Gothenburg, Sweden (Recruiting)
- St Thomas' Hospital — London, United Kingdom (Recruiting)
- Great Ormond Street Hospital — London, United Kingdom (Recruiting)
- Manchester Royal Infirmary — Manchester, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Reference Study ID Number: WP45335 https://forpatients.roche.com/
- Email: global-roche-genentech-trials@gene.com
- Phone: 888-662-6728 (U.S. Only)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.