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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Von Willebrand Factor Concentrates | Drug | Used according to local labeling or local treatment guidelines. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Bleed Rate (ABR) for Treated Bleeds | From Baseline to at least 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| ABR for All Bleeds | From Baseline to at least 24 weeks | |
| ABR for Treated Spontaneous Bleeds | From Baseline to at least 24 weeks | |
| ABR for Treated Joint Bleeds |
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Inclusion Criteria:
Exclusion Criteria:
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This study will observe approximately 40 participants with Type 3 VWD (aged 2 years and older) that are currently being treated with SOC prophylactic therapy.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis | Sacramento | California | 95817 | United States | ||
| University of Minnesota Medical Center |
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| Label | URL |
|---|---|
| Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours. | View source |
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| Von Willebrand Factor Concentrates and Factor VIII Concentrates | Drug | Used according to local labeling or local treatment guidelines. |
|
| Factor VIII Concentrates | Drug | Used according to local labeling or local treatment guidelines. |
|
| Recombinant Activated Factor VII | Drug | Used according to local labeling or local treatment guidelines. |
|
| Activated Prothrombin Complex Concentrate | Drug | Used according to local labeling or local treatment guidelines. |
|
| From Baseline to at least 24 weeks |
| Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale | From Baseline until study completion (at least 24 weeks) |
| Minneapolis |
| Minnesota |
| 55455 |
| United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| UZ Leuven Gasthuisberg | Leuven | 3000 | Belgium |
| The Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| McGill University Health Center | Montreal | Quebec | H4A 3J1 | Canada |
| IPS SURA Industriales Medellín | Medellín | Colombia |
| Hopital Claude Huriez - CHU Lille | Lille | 59037 | France |
| Groupe Hospitalier Necker Enfants Malades | Paris | 75015 | France |
| Universitätsklinikum Bonn | Bonn | 53127 | Germany |
| Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz | Duisburg | 47051 | Germany |
| Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin | Frankfurt/M. | 60590 | Germany |
| Universita' Degli Studi La Sapienza-Ist.Di Ematologia | Rome | Lazio | 00161 | Italy |
| IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Lombardy | 20122 | Italy |
| AOU Careggi | Florence | Tuscany | 50134 | Italy |
| Kurume University Hospital | Fukuoka | 830-0011 | Japan |
| Tokyo Medical University Hospital | Tokyo | 160-0023 | Japan |
| Erasmus MC | Rotterdam | 3015 GD | Netherlands |
| Instytut Hematologii i Transfuzjologii | Warsaw | 02-776 | Poland |
| Charlotte Maxeke Johannesburg Academic Hospital | Johannesburg | 2193 | South Africa |
| Hospital Universitario la Paz | Madrid | 28046 | Spain |
| Hospital Universtiario Virgen del Rocio | Seville | 41013 | Spain |
| Sahlgrenska Universitetssjukhuset | Gothenburg | S-413 45 | Sweden |
| St Thomas' Hospital | London | SE1 7EH | United Kingdom |
| Great Ormond Street Hospital | London | WC1N 3JH | United Kingdom |
| Manchester Royal Infirmary | Manchester | M13 9WL | United Kingdom |
| ID | Term |
|---|---|
| D056729 | von Willebrand Disease, Type 3 |
| ID | Term |
|---|---|
| D014842 | von Willebrand Diseases |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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