A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease… (NCT05695560) | Clinical Trial Compass
CompletedNot Applicable
A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
Canada12 participantsStarted 2023-02-24
Plain-language summary
The main aim of this study is to describe the experience and unmet needs of persons living with VWD and their caregivers in Canada.
The survey is planned to be done in two phases: The first phase will be directed at adult participants; the second phase will focus on children and teenagers. At the end of the first phase the Sponsor will decide if the second phase will be started.
Participants and their caregivers will be asked to answer a set of questions either using an online questionnaire or through interviews. The participant/caregiver's perception, experience, satisfaction, and unmet needs, and need for new treatments or new indications will be determined based on their responses to the questions.
Who can participate
Age range
0 Years – 50 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:
* Phase 1:
* Adult participants (age ≥18 years) with severe VWD (self-BAT ≥10)
* Participants who have received von Willebrand factor (VWF) treatment, either for on-demand treatment, regular prophylaxis, or situational prophylaxis (e.g., surgery) within the last 5 years
* For caregivers: Current caregiver of participants with severe VWD
* For caregivers and participants:
* Fluent in English or French
* Consent to participate in an individual phone interview and to fill self-administered questionnaires
* Additional inclusion criteria for virtual focus groups:
* Access to technology (Internet and email)
* Consent to participate in a virtual focus group with an audio recording of the session.
* Phase 2:
* Same as above for participants pediatric participants (age \<18 years) with severe VWD (self-PBQ score of ≥3 for at least one symptom).
Exclusion criteria:
* Phase 1:
* Participants or caregivers of participants who do not have severe symptoms of VWD
* Pediatric participants (age \<18 years)
* Participants who have not received any treatment (on-demand, regular or situational prophylaxis) within the last five years
* Participants who are successfully treated with desmopressin or anti-fibrinolytic medications
* Participants with inherited or acquired hemostatic or bleeding disorders other than congenital VWD (self-reported)
* Participants and caregivers of participants who show cognitive impairment (as assessed by the research nurse at t…
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
1
Number of Participants Categorized Based on Bleeding Characteristics
Timeframe: Up to approximately 9 months
2
Number of Participants Categorized by Impact on Daily Life
Timeframe: Up to approximately 9 months
3
Number of Participants Categorized Based on Disease Management