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Completed

NCT Number: NCT05695560

A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers

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.

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Key information

Conditions

Von Willebrand Disease (VWD) Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders von Willebrand Diseases

Age range

0 year–50 year

Sex eligibility

All sexes

Study type

Observational

Primary location

YolaRx Consultants

Montreal, H3W 1Y7, Canada

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

No intervention

Other

As this is an observational study, no intervention will be administered.

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

Secondary outcomes

  1. Time (Delay) to Treatment Initiation

    Time frame: Up to approximately 9 months

  2. Duration of Therapy Schedule

    Time frame: Up to approximately 9 months

  3. Number of Participants With Change in Treatment Frequency

    Time frame: Up to approximately 9 months

  4. Number of Participants With Bleed Control

    Time frame: Up to approximately 9 months

  5. Time to Bleed Control

    Time frame: Up to approximately 9 months

  6. Number of Participants Who Missed Days at Work/School

    Time frame: Up to approximately 9 months

    The number of participants who missed days at work/school will include categories for patient and caregiver.

  7. Duration of Inpatient and Outpatient Hospital Visits

    Time frame: Up to approximately 9 months

  8. Participant's Experience Assessed as Number of Participants Categorized Based on Symptom Severity and Comorbidities Over Time

    Time frame: Up to approximately 9 months

  9. Treatment Experience Based on Number of Participants Satisfied With the Treatment

    Time frame: Up to approximately 9 months

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

Unmet Needs of Patients Living With Von Willebrand Disease and Their Caregivers: Qualitative Survey on Current Standard of Care in Canada

Important dates

Study start
2023
Primary completion
2023
Study completion
2023
First posted
Jan 25, 2023
Registry last updated
Dec 20, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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