BetterLife FSHD: a patient-driven health platform
BetterLife FSHD: A Patient-driven Health and Research Platform
This project tries to connect people with FSHD to personalized resources and collect their health experiences so they can be matched to relevant studies and help improve care.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 5000 (estimated) |
| Ages | 1 Year and up |
| Sex | All |
| Sponsor | FSHD Society Academic / other |
| Locations | 1 site (Randolph, Massachusetts) |
| Trial ID | NCT07409142 on ClinicalTrials.gov |
What this trial studies
BetterLife FSHD is a patient-driven registry that securely collects de-identified health and experience data from people living with facioscapulohumeral muscular dystrophy. Participants complete short surveys on diagnosis, progression, management strategies, and quality-of-life topics on a quarterly and yearly schedule and receive a personalized feed of resources from the FSHD Society. Consent-based data are used to notify participants about clinical trial and research opportunities and to share de-identified datasets with approved researchers, industry, and regulators via a steering committee. Enrollment is open to US residents aged one year and older with a clinical or genetic diagnosis of FSHD or symptomatic individuals with a family history of FSHD.
Who should consider this trial
Good fit: Ideal candidates are people aged one year or older who live in the United States or its territories, have a clinical or genetic diagnosis of FSHD or a family history with symptoms, and can provide consent or assent with parental permission as applicable.
Not a fit: People living outside the United States or its territories or those unwilling to provide consent or share health information are unlikely to benefit from or participate in this platform.
Why it matters
Potential benefit: If successful, BetterLife could speed identification of eligible participants for trials and give researchers larger real-world datasets to guide improved care and new treatments for FSHD.
How similar studies have performed: Patient registries for rare neuromuscular diseases have previously helped accelerate trial recruitment and natural history research, so the registry approach is established rather than entirely novel.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Randolph, Massachusetts
- FSHD Society — Randolph, Massachusetts, United States (Recruiting)
Study contacts
- Principal investigator: Amanda Hill, MBA — FSHD Society
- Study coordinator: Kayleigh Worek, MS
- Email: betterlife@fshdsociety.org
- Phone: 781-301-6060
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.