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

NCT06883240 | Active Not Recruiting FDA Drug

• 1 other identifier: WP45335
• Study Type: observational
• Target: 44
• Locations: 14 countries
• Sites: 26

Brief 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.

Conditions

Von Willebrand Disease, Type 3

Outcome Measures

Primary Outcomes (1)

• Annualized Bleed Rate (ABR) for Treated Bleeds

  From Baseline to at least 24 weeks

Secondary Outcomes (4)

• 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

  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)

Study Arms (1)

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; Drug: Von Willebrand Factor Concentrates and Factor VIII Concentrates; Drug: Factor VIII Concentrates; Drug: Recombinant Activated Factor VII; Drug: Activated Prothrombin Complex Concentrate

Interventions

Factor VIII Concentrates DRUG

Used according to local labeling or local treatment guidelines.

Participants with Type 3 VWD Treated with Prophylactic SOC

Von Willebrand Factor Concentrates and Factor VIII Concentrates DRUG

Used according to local labeling or local treatment guidelines.

Participants with Type 3 VWD Treated with Prophylactic SOC

Activated Prothrombin Complex Concentrate DRUG

Used according to local labeling or local treatment guidelines.

Participants with Type 3 VWD Treated with Prophylactic SOC

Von Willebrand Factor Concentrates DRUG

Used according to local labeling or local treatment guidelines.

Participants with Type 3 VWD Treated with Prophylactic SOC

Recombinant Activated Factor VII DRUG

Used according to local labeling or local treatment guidelines.

Participants with Type 3 VWD Treated with Prophylactic SOC

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (26)

UC Davis

Sacramento, California, 95817, United States

Location

University of Minnesota Medical Center

Minneapolis, Minnesota, 55455, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

UZ Leuven Gasthuisberg

Leuven, 3000, Belgium

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

McGill University Health Center

Montreal, Quebec, H4A 3J1, Canada

Location

IPS SURA Industriales Medellín

Medellín, Colombia

Location

Hopital Claude Huriez - CHU Lille

Lille, 59037, France

Location

Groupe Hospitalier Necker Enfants Malades

Paris, 75015, France

Location

Universitätsklinikum Bonn

Bonn, 53127, Germany

Location

Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

Duisburg, 47051, Germany

Location

Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

Frankfurt/M., 60590, Germany

Location

Universita' Degli Studi La Sapienza-Ist.Di Ematologia

Rome, Lazio, 00161, Italy

Location

IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Lombardy, 20122, Italy

Location

AOU Careggi

Florence, Tuscany, 50134, Italy

Location

Kurume University Hospital

Fukuoka, 830-0011, Japan

Location

Tokyo Medical University Hospital

Tokyo, 160-0023, Japan

Location

Erasmus MC

Rotterdam, 3015 GD, Netherlands

Location

Instytut Hematologii i Transfuzjologii

Warsaw, 02-776, Poland

Location

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, 2193, South Africa

Location

Hospital Universitario la Paz

Madrid, 28046, Spain

Location

Hospital Universtiario Virgen del Rocio

Seville, 41013, Spain

Location

Sahlgrenska Universitetssjukhuset

Gothenburg, S-413 45, Sweden

Location

St Thomas' Hospital

London, SE1 7EH, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

Manchester Royal Infirmary

Manchester, M13 9WL, United Kingdom

Location

Related Links

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MeSH Terms

Conditions

von Willebrand Disease, Type 3

Condition Hierarchy (Ancestors)

von Willebrand Diseases; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 13, 2025
• First Posted: March 19, 2025
• Study Start: April 29, 2025
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: August 10, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1); ZA (1); ES (2); US (3); SE (1); GB (3); CO (1); JP (2); CA (2); IT (3); FR (2); DE (3); NL (1); PL (1)