Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- Patterns and Trends that Provide Evidence and Context for the Treatment Choices and Experiences of Those with Fabry Disease
研究概览
简要总结
The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.
详细描述
This will be a cross-sectional study conducted in approximately 130 individuals (or representative parents/caregivers of patients) living with Fabry disease. All study participants will complete the RSVP followed by a structured interview conducted by trained interviewers. It is estimated that each respondent will need up to 60 minutes for the entire process; 10 minutes to complete the RSVP including uploading the proof of Fabry disease diagnosis or verifying membership with Fabry groups, including but not limited to: Fabry Support and Information Group, National Fabry Disease Foundation, MPS Society UK, Morbus Fabry Selbsthilfergruppe, Fabry International Network, or others, and approximately 50 minutes to complete the interview.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be a person with Fabry disease who is 18 years or older or the parent/legal guardian of a living person with Fabry disease who is under the age of 18 years or who are 18 years and older who are unable to answer for themselves.
- •Confirmed diagnosis of Fabry disease with written proof of disease provided
- •Must have a genetic mutation that is amenable to oral therapy
- •Resident of Germany, the U.K or the U.S.
- •Able to read, write and communicate in German, or English.
- •Able to grant informed consent
- •Willing to participate in a 50 to 60-minute telephone interview, including follow up questions (if necessary) and information regarding adverse events (if necessary).
排除标准
- •Inability to meet any of the inclusion criteria
结局指标
主要结局
Patterns and Trends that Provide Evidence and Context for the Treatment Choices and Experiences of Those with Fabry Disease
时间窗: 1-2 months
The goal of the statistical analysis is to uncover patterns and trends that provide both evidence and context for the treatment choices and experiences of patients and families impacted by Fabry disease. All findings will be summarized in the final report, which will not identify any respondent as described above. At the conclusion of this study, the researchers may publish their findings in a medical / scientific journal.
次要结局
未报告次要终点