Clinical trial NCT05695560: A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
Official title: Unmet Needs of Patients Living With Von Willebrand Disease and Their Caregivers: Qualitative Survey on Current Standard of Care in Canada
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Von Willebrand Disease (VWD)
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2023-01-25
Enrollment: 12
Sex: ALL
Minimum age (years): 0
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants and caregivers of participants diagnosed with VWD in Canada.

Brief 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.

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.

Eligibility criteria: 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 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

Primary outcomes:
measure: Number of Participants Categorized Based on Bleeding Characteristics
timeFrame: Up to approximately 9 months
measure: Number of Participants Categorized by Impact on Daily Life
timeFrame: Up to approximately 9 months
measure: Number of Participants Categorized Based on Disease Management
timeFrame: Up to approximately 9 months
Secondary outcomes:
measure: Time (Delay) to Treatment Initiation
timeFrame: Up to approximately 9 months
measure: Duration of Therapy Schedule
timeFrame: Up to approximately 9 months
measure: Number of Participants With Change in Treatment Frequency
timeFrame: Up to approximately 9 months
measure: Number of Participants With Bleed Control
timeFrame: Up to approximately 9 months
measure: Time to Bleed Control
timeFrame: Up to approximately 9 months
measure: Number of Participants Who Missed Days at Work/School
timeFrame: Up to approximately 9 months
measure: Duration of Inpatient and Outpatient Hospital Visits
timeFrame: Up to approximately 9 months
measure: Participant's Experience Assessed as Number of Participants Categorized Based on Symptom Severity and Comorbidities Over Time
timeFrame: Up to approximately 9 months
measure: Treatment Experience Based on Number of Participants Satisfied With the Treatment
timeFrame: Up to approximately 9 months
Other outcomes:
Locations:
city: Montreal
country: Canada
facility: YolaRx Consultants
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: