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)
Official title: Vonicog Alfa (Recombinant Von Willebrand Factor) Treatment Outcomes in Von Willebrand Disease in the UK: a Retrospective Chart Review Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Von Willebrand Disease (VWD)
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2024-05-30
Enrollment: 34
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who have been diagnosed with congenital VWD and were prescribed rVWF in UK.

Brief 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.).


Eligibility criteria: 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.

Primary outcomes:
measure: 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
measure: Number of Surgical Procedures Between the Index Date and Following 12 Months
timeFrame: From index date up to 12 months
measure: 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
Secondary outcomes:
measure: Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)
timeFrame: Up to 24 months
measure: Number of Participants With Surgery Related Costs By Type
timeFrame: Up to 24 months
measure: Number of Participants With Bleeding Episodes Treated With rVWF
timeFrame: Up to 24 months
measure: Number of Surgical Procedures
timeFrame: Up to 24 months
measure: Number of Participants with Surgery Outcomes (Success, Failure, Complications)
timeFrame: Up to 24 months
Other outcomes:
Locations:
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospital
city: Liverpool
country: United Kingdom
facility: Liverpool University Hospital
city: London
country: United Kingdom
facility: Royal Free London
city: London
country: United Kingdom
facility: Imperial College Healthcare
city: Manchester
country: United Kingdom
facility: Manchester University
city: Oxford
country: United Kingdom
facility: Oxford University Hospital
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: