CompletedNot Applicable

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)

United Kingdom34 participantsStarted 2021-12-16

Plain-language summary

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.).

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: From index date up to 12 months

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

Timeframe: From index date up to 12 months

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

Timeframe: From index date up to 12 months

Trial details

NCT IDNCT06433778

SponsorTakeda

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-10-30

View on ClinicalTrials.gov More Von Willebrand Disease (VWD) trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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

Timeframe: From index date up to 12 months

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

Timeframe: From index date up to 12 months

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

Timeframe: From index date up to 12 months