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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
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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Observational | Other | Observational study |
| Measure | Description | Time Frame |
|---|---|---|
| Longitudinal Health Data | 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). | Assessed annually from enrollment until study completion (10 years) |
| Self Reported FSHD Progression | 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. | Assessed every 6 months, until study completion (10 years) |
| Measure | Description | Time Frame |
|---|---|---|
| Anxiety Patient Reported Outcome Measure | Assesses frequency and impact of anxiety symptoms using a 5-point rating system. Higher scores indicate more anxiety. | Quarterly, until study completion (10 years) |
| Depression Patient Reported Outcome Measure |
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Inclusion Criteria:
Exclusion Criteria:
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People with FSHD living in the United States or its territories age one year or older.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Kayleigh Worek, MS | Contact | 781-301-6060 | 2900 | betterlife@fshdsociety.org |
| Name | Affiliation | Role |
|---|---|---|
| Amanda Hill, MBA | FSHD Society | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| FSHD Society | Recruiting | Randolph | Massachusetts | 02368 | United States |
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| Label | URL |
|---|---|
| Learn more and enroll in BetterLife FSHD | View source |
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All individual participant data (IPD) collected by BetterLife is made available to the research community in the form of de-identified or limited datasets upon request and approval by the Steering Committee.
IPD and supporting information were available beginning August 2024 and with no planned end date.
Researchers can request access IPD and supporting information. Researchers may include pharmaceutical or biotechnology companies, academic institutions, hospitals, or other organizations involved in research and therapeutic development. Government agencies or payor systems who make decisions about treatment approvals and access, for example, the Food and Drug Administration (FDA) or the Institute for Clinical and Economic Review (ICER) can also request access.
To request access, researchers should visit www.BetterLifeFSHD.org or email BetterLife@FSHDSociety.org. Researchers must provide key details about their research project, including PI and study contacts, brief background information, clear study objectives, procedures, and outcomes, summary of importance for the field, and documentation of IRB approval, when required. All requests are reviewed by the Steering Committee. Projects that are approved must agree to a data use agreement.
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Assesses frequency and impact of depression symptoms using a 5-point rating system. Higher scores indicate more depression.
| Quarterly, until study completion (10 years) |
| Pain Patient Reported Outcome Measure | Assesses the quality of pain and interference with daily life using a 5-point rating system. Higher scores indicate higher levels of pain and interference. | Quarterly, until study completion (10 years) |
| Sleep Patient Reported Outcome Measure | Assesses the quality of sleep using a 5-point rating system. Higher scores indicate poor sleep quality. | Quarterly, until study completion (10 years) |
| Fatigue Patient Reported Outcome Measure | Assesses the presence and severity of fatigue using a 5-point rating system. Higher scores indicate more fatigue. | Quarterly, until study completion (10 years) |
| Upper Body Patient Reported Outcome Measure | Assesses the ability to perform activities of daily living with the upper body using a 5-point rating system. Higher scores indicate less difficulty completing tasks using the upper body. | Quarterly, until study completion (10 years) |
| Mobility Patient Reported Outcome Measure | Assesses the ability to perform activities with the lower body (i.e., standing up, sitting) using a 5-point rating system. Higher scores indicate less difficulty using the lower body. | Quarterly, until study completion (10 years) |
| Physical Activity Patient Reported Outcome Measure | Assesses the amount of time spent engaged in physical activity in minutes. Higher scores indicate greater levels of physical activity. | Quarterly, until study completion (10 years) |
| Falls Patient Reported Outcome Measure | Assesses fear of falling during daily activities using a 4-point scale. Higher scores indicate a greater fear of falling. | Quarterly, until study completion (10 years) |
| Research Preferences | Survey questions where participants rank their willingness to participate in various research procedures, concerns about participating in research trials, and preferences for remote/in-person/hybrid study visits. | Yearly until study completion (10 years) |
| Diagnostic Journey | Survey questions to describe participant experience and barriers getting diagnosed with FSHD. | One time at Baseline |
| Healthcare Experiences | Survey questions to describe healthcare utilization and experiences navigating healthcare settings. | Yearly, until study completion (10 years) |
| Women's Health | Survey questions about history of menstruation and pregnancy. | Yearly, until study completion (10 years) |
| Social Determinants of Health | Survey questions about current employment status/job security, neighborhood, religious practices, discrimination, and food security. | Yearly, until study completion (10 years) |
| ID | Term |
|---|---|
| D020391 | Muscular Dystrophy, Facioscapulohumeral |
| C563557 | Facioscapulohumeral Muscular Dystrophy 1B |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D057832 | Watchful Waiting |
| ID | Term |
|---|---|
| D017063 | Outcome Assessment, Health Care |
| D010043 | Outcome and Process Assessment, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
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