BetterLife FSHD: A Patient-driven Health and Research Platform
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Observational.
- Who it may be relevant to
- Registry conditions: FSH, FSH Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1. Basic parameters: from 1 year · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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
Detailed description
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.
Interventions
- Other Observational
Observational study
Primary outcome measures
- Longitudinal Health Data [Time frame: Assessed annually from enrollment until study completion (10 years)]
- Self Reported FSHD Progression [Time frame: Assessed every 6 months, until study completion (10 years)]
Secondary outcome measures (12)
- Anxiety Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Depression Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Pain Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Sleep Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Fatigue Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Upper Body Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Mobility Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Physical Activity Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Falls Patient Reported Outcome Measure [Time frame: Quarterly, until study completion (10 years)]
- Research Preferences [Time frame: Yearly until study completion (10 years)]
- Diagnostic Journey [Time frame: One time at Baseline]
- Healthcare Experiences [Time frame: Yearly, until study completion (10 years)]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- Individuals residing outside the United States or its territories
- Unable or unwilling to provide consent, or assent, when applicable
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- FSHD Society — Randolph
Identifiers
NCT: NCT07409142 · Pro00079388