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NCT Number: NCT06433778

A Study on the Outcomes of Recombinant Von Willebrand Factor on Demand Treatment and Prevention and Treatment of Bleeding During and After Surgery in Adults With Inherited Von Willebrand Disease in the United Kingdom (UK)

This study is a retrospective chart review study and will collect data on real world use of vonicog alfa (Recombinant Von Willebrand Factor [rVWF]). Von Willebrand disease (VWD) is the most common inherited bleeding disorder. rVWF is approved in Europe and UK to treat bleeding and to treat and prevent bleeding during surgeries in adults in 2018.

This study will review and collect information on the treatment and bleed prevention of adult persons with inherited VWD with rVWF in UK. These data were already collected as a part of the routine care.

The main aims of this study are to describe the use of rVWF in on-demand treatment of bleeding and the prevention of treatment and treatment of bleeding during surgeries. Other aims are to describe bleedings and their treatment as well as any surgeries before and after first treatment with rVWF and to gather information on the use of healthcare resources (such as hospital visits, emergency room visits, etc.).

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Key information

Conditions

Von Willebrand Disease (VWD) Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders von Willebrand Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University Hospitals Birmingham, Birmingham, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Adults (aged 18 or over at time of first administration of rVWF) who have provided informed consent and used rVWF within its licensed indication.
  • Participants who have been diagnosed with congenital von Willebrand disease.
  • Confirmed instance of
  • at least one bleed (either a new bleed or ongoing bleed treated under a treatment switch) treated on-demand with rVWF between 01-Oct-2020 and 30-Jun- 2022 and/or
  • treatment to prevent and treat surgical bleeds with rVWF between 01-Oct-2020 and 30-Jun-2022

Exclusion criteria

  • Participants who were aged 17 years or less at the time of the first administration of rVWF.
  • Participants who have been diagnosed with any other bleeding disorders or factor deficiencies including acquired von Willebrand disease.
  • Participants with neutralising antibodies/inhibitors to VWF.
  • Participants participation in a clinical trial of an investigational medical product during the study period.

Treatment and study plan

No intervention

Other

This is a non-interventional study.

Primary outcomes

  1. Number of Participants With Bleeding Episodes Treated with rVWF Between the Index Date and Following 12 Months

    Time frame: From index date up to 12 months

    Bleeding episodes will be assessed based on categories of overall and annualized, type, severity, location, bleed frequency, bleed type/location and bleed severity.

  2. Number of Surgical Procedures Between the Index Date and Following 12 Months

    Time frame: From index date up to 12 months

    Surgical procedures included surgery type, severity, category (emergency or elective). Data on the participants use of rVWF in the pre-, intra- and post-operative setting during the period between the index date and the following 12 months, will be stratified by VWD type, surgery type, severity, location, or treatment rationale.

  3. Number of Participants with Surgery Outcomes (Success, Failure, Complications) Between the Index Date and Following 12 Months

    Time frame: From index date up to 12 months

    Surgery outcomes (success, failure, complications) will be reported.

Secondary outcomes

  1. Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)

    Time frame: Up to 24 months

    HRU will include bleeding-related hospitalization rates, outpatient visits, accident and emergency visits.

  2. Number of Participants With Surgery Related Costs By Type

    Time frame: Up to 24 months

    Surgery related costs by type will include length of stay in intensive care unit (ICU), rVWF consumption, factor VIII (FVIII) consumption, VWD treatment consumption, laboratory tests and examinations.

  3. Number of Participants With Bleeding Episodes Treated With rVWF

    Time frame: Up to 24 months

    Bleeding episodes will be reported based on type, severity, location, treatment, and outcomes, of all recorded bleeds within 12 months prior to and 12 months following the first administration of rVWF.

  4. Number of Surgical Procedures

    Time frame: Up to 24 months

    Surgical procedures will be reported based on surgery type (orthopaedic, gastro-intestinal, dental, etc), severity (major, minor), category (emergency or elective) treatment duration and outcomes, of all recorded surgeries within 12 months prior to and 12 months following the first administration of rVWF.

  5. Number of Participants with Surgery Outcomes (Success, Failure, Complications)

    Time frame: Up to 24 months

    Surgery outcomes (success, failure, complications) based on average number of post-operative bleeds will be reported.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

Vonicog Alfa (Recombinant Von Willebrand Factor) Treatment Outcomes in Von Willebrand Disease in the UK: a Retrospective Chart Review Study

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
May 30, 2024
Registry last updated
Mar 11, 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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