## Protocol Section
### Identification Module
**NCT ID:** NCT06433778

**Brief Title:** 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

#### Organization Study ID Info
**ID:** TAK-577-5001
#### Organization
**Class:** INDUSTRY
**Full Name:** Takeda
### Status Module
#### Completion Date
**Date:** 2023-10-30

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-30

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-23

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2023-10-30

**Type:** ACTUAL

#### Start Date
**Date:** 2021-12-16

**Type:** ACTUAL

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-30
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-23
**Study First Submit QC Date:** 2024-05-23
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Takeda

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** False

**Oversight Has DMC:** False

### Description Module

**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.).

### Conditions Module

**Conditions:**

- Von Willebrand Disease (VWD)

**Keywords:**

- Drug Therapy

### Design Module

#### Design Info

**Observational Model:** COHORT

**Time Perspective:** RETROSPECTIVE

#### Enrollment Info

**Count:** 34

**Type:** ACTUAL

**Study Type:** OBSERVATIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** 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.

**Intervention Names:**

- Other: No Intervention

**Label:** Participants diagnosed With Congenital VWD

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Participants diagnosed With Congenital VWD

**Description:** This is a non-interventional study.

**Name:** No Intervention

**Type:** OTHER

### Outcomes Module

#### Primary Outcomes

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

**Measure:** 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

**Description:** 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.

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

**Time Frame:** From index date up to 12 months

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

**Measure:** 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

#### Secondary Outcomes

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

**Measure:** Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)

**Time Frame:** Up to 24 months

**Description:** 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.

**Measure:** Number of Participants With Surgery Related Costs By Type

**Time Frame:** Up to 24 months

**Description:** 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.

**Measure:** Number of Participants With Bleeding Episodes Treated With rVWF

**Time Frame:** Up to 24 months

**Description:** 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.

**Measure:** Number of Surgical Procedures

**Time Frame:** Up to 24 months

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

**Measure:** Number of Participants with Surgery Outcomes (Success, Failure, Complications)

**Time Frame:** Up to 24 months

### Eligibility Module

**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.

**Minimum Age:** 18 Years

**Sampling Method:** NON_PROBABILITY_SAMPLE

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

**Study Population:** Participants who have been diagnosed with congenital VWD and were prescribed rVWF in UK.

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Birmingham

**Country:** United Kingdom

**Facility:** University Hospitals Birmingham

**Zip:** B15 2GW

**Location 2:**

**City:** Leeds

**Country:** United Kingdom

**Facility:** Leeds Teaching Hospital

**Zip:** LS9 7TF

**Location 3:**

**City:** Liverpool

**Country:** United Kingdom

**Facility:** Liverpool University Hospital

**Zip:** L7 8XP

**Location 4:**

**City:** London

**Country:** United Kingdom

**Facility:** Royal Free London

**Zip:** NW3 2QG

**Location 5:**

**City:** London

**Country:** United Kingdom

**Facility:** Imperial College Healthcare

**Zip:** W2 1NY

**Location 6:**

**City:** Manchester

**Country:** United Kingdom

**Facility:** Manchester University

**Zip:** M13 9WL

**Location 7:**

**City:** Oxford

**Country:** United Kingdom

**Facility:** Oxford University Hospital

**Zip:** OX3 9DU

#### Overall Officials

**Official 1:**

**Affiliation:** Takeda

**Name:** Study Director

**Role:** STUDY_DIRECTOR

### IPD Sharing Statement Module

**Access Criteria:** IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

**Description:** Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

**Info Types:**

- STUDY_PROTOCOL

- SAP

- ICF

- CSR

**IPD Sharing:** YES

**URL:** https://vivli.org/ourmember/takeda/

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000025861

- Term: Blood Coagulation Disorders, Inherited

- ID: D000001778

- Term: Blood Coagulation Disorders

- ID: D000006402

- Term: Hematologic Diseases

- ID: D000020147

- Term: Coagulation Protein Disorders

- ID: D000001791

- Term: Blood Platelet Disorders

- ID: D000006474

- Term: Hemorrhagic Disorders

- ID: D000030342

- Term: Genetic Diseases, Inborn

### Condition Browse Module - Browse Branches

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

- Abbrev: BC15

- Name: Blood and Lymph Conditions

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: BC14

- Name: Heart and Blood Diseases

### Condition Browse Module - Browse Leaves

- ID: M9556

- Name: Hemorrhage

- Relevance: LOW

- As Found: Unknown

- ID: M17585

- Name: Von Willebrand Diseases

- Relevance: HIGH

- As Found: Von Willebrand Disease

- ID: M21977

- Name: Hemostatic Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M5059

- Name: Blood Coagulation Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M23095

- Name: Blood Coagulation Disorders, Inherited

- Relevance: LOW

- As Found: Unknown

- ID: M9490

- Name: Hematologic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M21982

- Name: Coagulation Protein Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M5072

- Name: Blood Platelet Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M9560

- Name: Hemorrhagic Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000014842

- Term: Von Willebrand Diseases

### Misc Info Module

- Version Holder: 2024-05-31
