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NCT Number: NCT06883240

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

UZ Leuven Gasthuisberg, Leuven, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Von Willebrand Factor Concentrates

Drug

Used according to local labeling or local treatment guidelines.

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.

Primary outcomes

  1. Annualized Bleed Rate (ABR) for Treated Bleeds

    Time frame: From Baseline to at least 24 weeks

Secondary outcomes

  1. ABR for All Bleeds

    Time frame: From Baseline to at least 24 weeks

  2. ABR for Treated Spontaneous Bleeds

    Time frame: From Baseline to at least 24 weeks

  3. ABR for Treated Joint Bleeds

    Time frame: From Baseline to at least 24 weeks

  4. Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale

    Time frame: From Baseline until study completion (at least 24 weeks)

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study title

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

Acronym: WILL-EMI NIS

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Mar 19, 2025
Registry last updated
Jul 10, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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