Unmet Needs of Patients Living With Von Willebrand Disease and Their Caregivers: Qualitative Survey on Current Standard of Care in Canada
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Number of Participants Categorized by Impact on Daily Life
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 0 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Number of Participants Categorized by Impact on Daily Life
时间窗: 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 Bleeding Characteristics
时间窗: Up to approximately 9 months
Bleeding characteristics will include categories of bleed frequency, bleed type/location and bleed severity.
Number of Participants Categorized Based on Disease Management
时间窗: 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.
次要结局
- Time (Delay) to Treatment Initiation(Up to approximately 9 months)
- Number of Participants Who Missed Days at Work/School(Up to approximately 9 months)
- Duration of Therapy Schedule(Up to approximately 9 months)
- Participant's Experience Assessed as Number of Participants Categorized Based on Symptom Severity and Comorbidities Over Time(Up to approximately 9 months)
- Number of Participants With Change in Treatment Frequency(Up to approximately 9 months)
- Time to Bleed Control(Up to approximately 9 months)
- Treatment Experience Based on Number of Participants Satisfied With the Treatment(Up to approximately 9 months)
- Number of Participants With Bleed Control(Up to approximately 9 months)
- Duration of Inpatient and Outpatient Hospital Visits(Up to approximately 9 months)