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

Recruiting

• Conditions: Von Willebrand Disease, Type 3
• Interventions: 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

• Registration Number: NCT06883240
• Lead Sponsor: Hoffmann-La Roche

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-03-13
• Study First Submitted QC: 2025-03-13
• Study First Posted: 2025-03-19
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-16
• Last Update Posted: 2025-05-18
• Study Start: 2025-05-27
• Primary Completion: 2026-11-01
• Study Completion: 2026-11-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

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

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Participants with Type 3 VWD Treated with Prophylactic SOC	Von Willebrand Factor Concentrates	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.
Participants with Type 3 VWD Treated with Prophylactic SOC	Von Willebrand Factor Concentrates and Factor VIII Concentrates	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.
Participants with Type 3 VWD Treated with Prophylactic SOC	Recombinant Activated Factor VII	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.
Participants with Type 3 VWD Treated with Prophylactic SOC	Factor VIII Concentrates	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.
Participants with Type 3 VWD Treated with Prophylactic SOC	Activated Prothrombin Complex Concentrate	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.

Primary Outcome Measures

Name	Time	Method
Annualized Bleed Rate (ABR) for Treated Bleeds	From Baseline to at least 24 weeks

Secondary Outcome Measures

Name	Time	Method
ABR for All Bleeds	From Baseline to at least 24 weeks
ABR for Treated Spontaneous 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)
ABR for Treated Joint Bleeds	From Baseline to at least 24 weeks

Trial Locations

Locations (8)

UZ Leuven Gasthuisberg; 🇧🇪 Leuven, Belgium

Universita' Degli Studi La Sapienza-Ist.Di Ematologia; 🇮🇹 Roma, Lazio, Italy

AOU Careggi; 🇮🇹 Firenze, Toscana, Italy

Kurume University Hospital; 🇯🇵 Fukuoka, Japan

IPS SURA Industriales Medellín; 🇨🇴 Medellin, Colombia

Hospital Universitario la Paz; 🇪🇸 Madrid, Spain

Hospital Universitario Virgen del Rocio; 🇪🇸 Sevilla, Spain

Great Ormond Street Hospital; 🇬🇧 London, United Kingdom