- ICH GCP
- US Clinical Trials Registry
- Clinical Trial 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)
Vonicog Alfa (Recombinant Von Willebrand Factor) Treatment Outcomes in Von Willebrand Disease in the UK: a Retrospective Chart Review Study
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.).
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Birmingham, United Kingdom, B15 2GW
- University Hospitals Birmingham
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Leeds, United Kingdom, LS9 7TF
- Leeds Teaching Hospital
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Liverpool, United Kingdom, L7 8XP
- Liverpool University Hospital
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London, United Kingdom, NW3 2QG
- Royal Free London
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London, United Kingdom, W2 1NY
- Imperial College Healthcare
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Manchester, United Kingdom, M13 9WL
- Manchester University
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Oxford, United Kingdom, OX3 9DU
- Oxford University Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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.
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
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Participants diagnosed With Congenital VWD
Participants who have been diagnosed with congenital VWD and prescribed rVWF within the index date range (defined as the first administration of rVWF and must fall between 1st October 2020 and 30th June 2022) will be assessed using data obtained from medical records to evaluate the treatment outcome of rVWF in real-world clinical practice.
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This is a non-interventional study.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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Bleeding episodes will be assessed based on categories of overall and annualized, type, severity, location, bleed frequency, bleed type/location and bleed severity.
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From index date up to 12 months
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Number of Surgical Procedures Between the Index Date and Following 12 Months
Time Frame: From index date up to 12 months
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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.
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From index date up to 12 months
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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
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Surgery outcomes (success, failure, complications) will be reported.
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From index date up to 12 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)
Time Frame: Up to 24 months
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HRU will include bleeding-related hospitalization rates, outpatient visits, accident and emergency visits.
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Up to 24 months
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Number of Participants With Surgery Related Costs By Type
Time Frame: Up to 24 months
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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.
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Up to 24 months
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Number of Participants With Bleeding Episodes Treated With rVWF
Time Frame: Up to 24 months
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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.
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Up to 24 months
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Number of Surgical Procedures
Time Frame: Up to 24 months
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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.
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Up to 24 months
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Number of Participants with Surgery Outcomes (Success, Failure, Complications)
Time Frame: Up to 24 months
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Surgery outcomes (success, failure, complications) based on average number of post-operative bleeds will be reported.
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Up to 24 months
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Study Director, Takeda
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- TAK-577-5001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Access Criteria
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
- ICF
- CSR
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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