BetterLife FSHD: A Patient-driven Health and Research Platform
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 5,000
- 试验地点
- 1
- 主要终点
- Longitudinal Health Data
研究概览
简要总结
BetterLife FSHD is a registry platform built to support people living with FSHD. It connects patients with personalized resources, tools, and research opportunities that match their health experiences and needs. At the same time, BetterLife collects secure health and experience data from patients to support research. This data is shared with researchers to help better understand FSHD and work toward improved care, treatments, and outcomes for the community.
Learn more and enroll at: www.BetterLifeFSHD.org
详细描述
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder that causes relentless weakening of skeletal muscles. BetterLife FSHD is a patient-driven health platform and research registry that aims to help FSHD patients live their best lives while also powering research.
In BetterLife, participants are prompted to respond to a series of short surveys spread out over a quarterly and yearly basis. Survey topics include demographics, health history, FSHD diagnosis and progression, FSHD management strategies, and quality of life domains like pain, fatigue, and mental health.
As participants provide survey data, they receive a personalized feed of resources relevant to them from the FSHD Society's library of articles, blogs, videos, and webinars. Survey data is also used to inform participants which clinical trials and other research studies they may be eligible for.
The information that BetterLife FSHD collects is stored and managed in a modern and secure real-world data infrastructure. De-identified data is made available upon request to researchers, clinicians, biopharmaceutical companies, regulator/payor bodies, and other organizations involved in FSHD research and therapeutic development with approval from a steering committee. BetterLife can also be used to conduct and support research initiatives external to the FSHD Society.
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
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals with a clinical or genetic diagnosis of FSHD, or individuals with a family history of FSHD who are showing symptoms
- •Age 1 year or older
- •Residing in the United States or its territories
- •If age 18 or older, individual must be able and willing to provide consent
- •If under age 18, individual must be able and willing to provide assent, when applicable, and have a parent or legal guardian register and provide consent
排除标准
- •Individuals residing outside the United States or its territories
- •Unable or unwilling to provide consent, or assent, when applicable
结局指标
主要结局
Longitudinal Health Data
时间窗: Assessed annually from enrollment until study completion (10 years)
Survey questions to capture information on demographics, health history, FSHD diagnosis (clinical diagnosis, genetic testing, family history, etc.), and FSHD symptom management (e.g., use of assistive devices, ventilation, surgery).
Self Reported FSHD Progression
时间窗: Assessed every 6 months, until study completion (10 years)
Survey questions that cover muscle weakness, symptom onset, and key progression milestones, and which can be used to estimate FSHD Clinical Score (FCS) . A higher score indicates more disease severity.
次要结局
- Anxiety Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Depression Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Pain Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Sleep Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Fatigue Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Upper Body Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Mobility Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Physical Activity Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Falls Patient Reported Outcome Measure(Quarterly, until study completion (10 years))
- Research Preferences(Yearly until study completion (10 years))
- Diagnostic Journey(One time at Baseline)
- Healthcare Experiences(Yearly, until study completion (10 years))
- Women's Health(Yearly, until study completion (10 years))
- Social Determinants of Health(Yearly, until study completion (10 years))
研究者
Amanda Hill
Senior Director of Research
FSHD Society