The Lived Experience of People With Von Willebrand Disease (VWD360)

March 12, 2024 updated by: Haemnet

von Willebrand disease (vWD) is reported to be the most common bleeding disorder, with prevalence estimated at 1% of the general population. Despite this, little is known about its natural history, or of the impact it has on affected individuals and their families.

The Haemnet vWD360 programme is a mixed-methods, natural history study designed to gain a greater understanding of vWD and its impact on individuals and their families. It comprises both qualitative and quantitative approaches and is designed to include the perspectives of individuals with a diagnosis of any subtype of vWD.

The vWD360 study includes three components:

  • Quantitative cross-sectional survey
  • Qualitative one-to-one interviews with affected individuals
  • 30-day bleed diary.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

von Willebrand Disease (vWD) is an inherited blood clotting (coagulation) disorder characterized by a reduction (quantitative) or poor function (qualitative) defect of factor VIII (FVIII) and/or von Willebrand factor (vWF). There are numerous subtypes categorised as:

  • Type 1: a quantitative defect characterised by decreased levels of vWF in the circulation. Many affected individuals have a mild bleeding phenotype but may have heavy menstrual bleeding (HMB) and bleed following trauma/surgery.
  • Type 2: a qualitative defect that is further divided into four subtypes
  • Type 2A - vWF is unable to bind to form the large vWF multimers required for coagulation
  • Type 2B - vWF has enhanced binding to platelet glycoprotein Ib (GPIb), causing rapid clearance of platelets and an associated thrombocytopenia
  • Type 2M - vWF has a decreased ability to bind to GPIb
  • Type 2N - there is a deficiency of vWF binding to FVIII
  • Type 3: the most severe vWD subtype, characterised by complete absence of vWF production and an inability to bind with FVIII, resulting in a severely reduced FVIII level.

Acquired vWD can develop as an autoimmune disorder, as a result of cancer, some cardiac conditions or following of certain drugs. It will not be considered as part of this study.vWD is characterized by prolonged or spontaneous bleeding from birth. Affected individuals tend to bruise easily, may have frequent nosebleeds (epistaxis), may bleed from the gums, bleeding within tissues (haematoma), in the gastrointestinal tract (more common later in life) and joint bleeds (in Type 3). vWD causes prolonged bleeding following injury, trauma, or surgery (including dental work). Women with vWD can have prolonged heavy menstrual bleeding, they may also have an increased risk of excessive blood loss during pregnancy and childbirth.

The severity and frequency of the bleeding episodes in vWD can vary greatly among affected individuals, even within the same family. The bleeding phenotype correlates to some degree with the subtype of VWD, with those with the severest form (Type 3) having the most bleeding.

Treatment varies based on the diagnosis. In Types 1 and 2 vWD treatment is usually 'on-demand' (after bleeding occurs) with some patients receiving prophylaxis if they have significant frequent bleeding. On demand treatment may be with oral, intra-nasal or subcutaneous treatments or with intravenous infusions of clotting factor concentrates containing FVIII/vWF. This is the method of treatment for all bleeding and prophylaxis in Type 3 vWD, where for some patients, treatment may be given at home.

The lack of routine prophylaxis in Type 1 and 2 vWD means that most patients are reliant on hospital delivered care, which may involve frequent clinic appointments, causing prolonged bleeding due to a lack of timely administration of treatment. This can result in concurrent illnesses such as iron deficiency anaemia, which further impacts on the quality of life of affected individuals and their families.

There remains a need for a comprehensive understanding of the experience of people with vWD across the whole spectrum of subtypes in order to identify:

  • The nature and range of symptoms that people experience and how these vary with the different disease subtypes
  • The variability in pathways through which patients progress to access appropriate care
  • The impact of living with vWD on the individual's quality of life.

Study Type

Observational

Enrollment (Estimated)

500

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

People with a confirmed diagnosis of von Willebrands disease, aged 16 yrs and older (18 yrs and older in the US).

Description

Inclusion Criteria:

  • Adults aged 16 and above (UK and Ireland) and adults aged over 18 (in USA) with a confirmed diagnosis of inherited vWD of known diagnostic subtype and vWF level.
  • For the qualitative interview-based substudy, 30 adults who have completed the survey and who wish to be interviewed will be purposively selected for a broad range of ages and diagnostic subtype.
  • For the bleed diary substudy, 50 adults who have completed the survey and who wish to take part will be purposively selected for a broad range of ages and diagnostic subtype.

Exclusion Criteria:Participants will be excluded from the study if they:

  • Have acquired vWD
  • Have other inherited bleeding disorders
  • Do not wish to participate in or to consent to the study.

Those for whom written/spoken English would prohibit participation will also be excluded.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Survey Arm
All participants will be asked to complete an online survey
Other: Online Survey
All participants will be asked to complete an online survey
Interview Arm
A sample of the survey group will be asked to take part in a single qualitative interview
Other: Qualitative Interview
30 survey participants will take part in a single one-hour qualitative interview.
Bleed Diary Arm
A sample of the survey group will be asked to take part in a 30 day bleed diary
Other: Bleed Diary
50 participants will complete a 30 day bleed diary

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Bleeding Episodes
Time Frame: Survey - The month prior to completing the survey. Bleed Diary - 30 days from consent.
To identify differences in bleeding type and rate between sub-types of vWD
Survey - The month prior to completing the survey. Bleed Diary - 30 days from consent.
Pain experiences
Time Frame: Survey - The month prior to completion of the survey
To identify evidence of chronic and acute pain between sub-types of vWD
Survey - The month prior to completion of the survey
Daily Activities
Time Frame: Survey - one month prior to completion of the survey. Interview - life historical
To identify differences in daily activity between sub-types of vWD
Survey - one month prior to completion of the survey. Interview - life historical

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Haemnet

Collaborators

Hemab ApS

Investigators

  • Principal Investigator: Simon Fletcher, MA, Haemnet

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 8, 2023

Primary Completion (Estimated)

December 31, 2024

Study Completion (Estimated)

December 31, 2024

Study Registration Dates

First Submitted

September 26, 2023

First Submitted That Met QC Criteria

September 26, 2023

First Posted (Actual)

October 3, 2023

Study Record Updates

Last Update Posted (Actual)

March 13, 2024

Last Update Submitted That Met QC Criteria

March 12, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • v1.0 dated 15 June 2023

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

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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