Von Willebrand Disease (VWD)NCT05695560
A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
An observational study of Von Willebrand Disease (VWD), run by Takeda at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 50 years
- Conditions the study is for
- Von Willebrand Disease (VWD)
- How many people
- 12 participants
- Where
- 1 siteCanada
- Study dates
- 24 Feb 2023to 30 Nov 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants and caregivers of participants diagnosed with VWD in Canada.
Who may be able to join
- 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
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- 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).
Who cannot join
- 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)
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- Participants and caregivers of participants who show cognitive impairment (as assessed by the research nurse at the time of screening)
- Participants and caregivers who are qualified as health care practitioners currently working in a health-care capacity (e.g., physician, nurse, or healthcare aid)
- Participants or caregivers who do not reside in Canada
- Current or past participation within the last 12 months in a clinical trial
- Phase 2: Same as above excluding participants of ≥18 years of age
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Unmet Needs of Patients Living With Von Willebrand Disease and Their Caregivers: Qualitative Survey on Current Standard of Care in Canada
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.
Read the detailed description
This study is a non-interventional, prospective, qualitative survey to know the unmet needs of participants living with VWD and their caregivers.
The study will enroll approximately 49 patients, taking into scope both the participant's and caregiver's perspectives, and is planned to be conducted in two phases:
Phase 1: Adult Participants Phase 2: Pediatric Participants The decision to proceed with Phase 2 will be determined at the completion of Phase 1.
This multi-center trial will be conducted in Canada. The overall time for data collection in this study is approximately 9 months.
- Conditions
- Von Willebrand Disease (VWD)
- Being tested
- No Intervention
- Keywords
- Drug Therapy
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
As this is an observational study, no intervention will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: Adult Participants | Adult participants with severe VWD (self-bleeding assessment tool [BAT] score ≥10) and their caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform. Receives: Other: No Intervention | |
| Phase 2: Pediatric Participants | Pediatric participants with severe VWD (self-pediatric bleeding questionnaire [PBQ] score of ≥3 for at least one symptom and caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform. The decision to proceed with Phase 2 will be determined following completion of Phase 1. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Categorized Based on Bleeding Characteristics
Bleeding characteristics will include categories of bleed frequency, bleed type/location and bleed severity.
Measured: Up to approximately 9 months
Number of Participants Categorized by Impact on Daily Life
The age-adapted impact on daily life will be categorized by questions related to quality of life, physical activity, professional life, school, financial impact, mental health, relationships, avoidance of social and physical activities, and impact on daily activities.
Measured: Up to approximately 9 months
Number of Participants Categorized Based on Disease Management
Disease management will be categorized by need for subsequent therapies, need for additional investigations, time needed for disease management, impact on future planning and treatment access for aging participants.
Measured: Up to approximately 9 months
Secondary outcomes (9)
Time (Delay) to Treatment Initiation
Measured: Up to approximately 9 months
Duration of Therapy Schedule
Measured: Up to approximately 9 months
Number of Participants With Change in Treatment Frequency
Measured: Up to approximately 9 months
Number of Participants With Bleed Control
Measured: Up to approximately 9 months
Time to Bleed Control
Measured: Up to approximately 9 months
Number of Participants Who Missed Days at Work/School
The number of participants who missed days at work/school will include categories for patient and caregiver.
Measured: Up to approximately 9 months
Duration of Inpatient and Outpatient Hospital Visits
Measured: Up to approximately 9 months
Participant's Experience Assessed as Number of Participants Categorized Based on Symptom Severity and Comorbidities Over Time
Measured: Up to approximately 9 months
Treatment Experience Based on Number of Participants Satisfied With the Treatment
Measured: Up to approximately 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2023
- Study start24 Feb 2023
- Primary completion30 Nov 2023
- Study completion30 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease (VWD)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05695560
- Sponsor's protocol ID
- TAK-577-4002
- Status last verified
- Dec 2023
- First submitted
- 13 Jan 2023
- Last updated
- 20 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 20 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Unmet Needs of Patients Living With Von Willebrand Disease and Their Caregivers: Qualitative Survey on Current Standard of Care in Canada
- ClinicalTrials.gov ID
- NCT05695560
- Acronym / sponsor ID
- TAK-577-4002
- Conditions
- Von Willebrand Disease (VWD)
- Keywords
- Drug Therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 24 Feb 2023 (actual)
- Primary completion
- 30 Nov 2023 (actual)
- Study completion
- 30 Nov 2023 (actual)
- First posted
- 25 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study is a non-interventional, prospective, qualitative survey to know the unmet needs of participants living with VWD and their caregivers.
The study will enroll approximately 49 patients, taking into scope both the participant's and caregiver's perspectives, and is planned to be conducted in two phases:
Phase 1: Adult Participants Phase 2: Pediatric Participants The decision to proceed with Phase 2 will be determined at the completion of Phase 1.
This multi-center trial will be conducted in Canada. The overall time for data collection in this study is approximately 9 months.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phase 1: Adult Participants | – | Other: No InterventionAdult participants with severe VWD (self-bleeding assessment tool [BAT] score ≥10) and their caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform. |
| Phase 2: Pediatric Participants | – | Other: No InterventionPediatric participants with severe VWD (self-pediatric bleeding questionnaire [PBQ] score of ≥3 for at least one symptom and caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform. The decision to proceed with Phase 2 will be determined following completion of Phase 1. |
Who may qualify
- Ages
- 0 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants and caregivers of participants diagnosed with VWD in Canada.
Key inclusion criteria (12 listed)
- 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:
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionAs this is an observational study, no intervention will be administered. | Other | Phase 1: Adult Participants, Phase 2: Pediatric Participants |
Primary outcome
Number of Participants Categorized Based on Bleeding Characteristics
Time frame: Up to approximately 9 months
Bleeding characteristics will include categories of bleed frequency, bleed type/location and bleed severity.
Number of Participants Categorized by Impact on Daily Life
Time frame: Up to approximately 9 months
The age-adapted impact on daily life will be categorized by questions related to quality of life, physical activity, professional life, school, financial impact, mental health, relationships, avoidance of social and physical activities, and impact on daily activities.
Number of Participants Categorized Based on Disease Management
Time frame: Up to approximately 9 months
Disease management will be categorized by need for subsequent therapies, need for additional investigations, time needed for disease management, impact on future planning and treatment access for aging participants.
9 secondary outcomes
Time (Delay) to Treatment Initiation
Time frame: Up to approximately 9 months
Duration of Therapy Schedule
Time frame: Up to approximately 9 months
Number of Participants With Change in Treatment Frequency
Time frame: Up to approximately 9 months
Number of Participants With Bleed Control
Time frame: Up to approximately 9 months
Time to Bleed Control
Time frame: Up to approximately 9 months
Number of Participants Who Missed Days at Work/School
Time frame: Up to approximately 9 months
Duration of Inpatient and Outpatient Hospital Visits
Time frame: Up to approximately 9 months
Participant's Experience Assessed as Number of Participants Categorized Based on Symptom Severity and Comorbidities Over Time
Time frame: Up to approximately 9 months
Treatment Experience Based on Number of Participants Satisfied With the Treatment
Time frame: Up to approximately 9 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Dec 2023.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease (VWD), run by Takeda at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Categorized Based on Bleeding Characteristics (time frame: Up to approximately 9 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Dec 2023.