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NCT Number: NCT07409142

BetterLife FSHD: A Patient-driven Health and Research Platform

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

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Key information

Conditions

FSH Congenital, Hereditary, and Neonatal Diseases and Abnormalities FSH Muscular Dystrophy FSHD FSHD - Facioscapulohumeral Muscular Dystrophy FSHD1 FSHD2 Facioscapulohumeral Muscular Dystrophy Facioscapulohumeral Muscular Dystrophy (FSHD) Facioscapulohumeral Muscular Dystrophy 1 Facioscapulohumeral Muscular Dystrophy 1B Facioscapulohumeral Muscular Dystrophy 2 Genetic Diseases, Inborn Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Facioscapulohumeral Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

1 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

FSHD Society

Randolph, Massachusetts, 02368, United States

Location status: Recruiting

Location contact

Kayleigh Worek, Program Manager, MS

CONTACT

[email protected]

781-301-6060 ext. 2900

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Observational

Other

Observational study

Primary outcomes

  1. Longitudinal Health Data

    Time frame: 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).

  2. Self Reported FSHD Progression

    Time frame: 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.

Secondary outcomes

  1. Anxiety Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    Assesses frequency and impact of anxiety symptoms using a 5-point rating system. Higher scores indicate more anxiety.

  2. Depression Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    Assesses frequency and impact of depression symptoms using a 5-point rating system. Higher scores indicate more depression.

  3. Pain Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    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.

  4. Sleep Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    Assesses the quality of sleep using a 5-point rating system. Higher scores indicate poor sleep quality.

  5. Fatigue Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    Assesses the presence and severity of fatigue using a 5-point rating system. Higher scores indicate more fatigue.

  6. Upper Body Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    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.

  7. Mobility Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    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.

  8. Physical Activity Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    Assesses the amount of time spent engaged in physical activity in minutes. Higher scores indicate greater levels of physical activity.

  9. Falls Patient Reported Outcome Measure

    Time frame: Quarterly, until study completion (10 years)

    Assesses fear of falling during daily activities using a 4-point scale. Higher scores indicate a greater fear of falling.

  10. Research Preferences

    Time frame: Yearly until study completion (10 years)

    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.

  11. Diagnostic Journey

    Time frame: One time at Baseline

    Survey questions to describe participant experience and barriers getting diagnosed with FSHD.

  12. Healthcare Experiences

    Time frame: Yearly, until study completion (10 years)

    Survey questions to describe healthcare utilization and experiences navigating healthcare settings.

  13. Women's Health

    Time frame: Yearly, until study completion (10 years)

    Survey questions about history of menstruation and pregnancy.

  14. Social Determinants of Health

    Time frame: Yearly, until study completion (10 years)

    Survey questions about current employment status/job security, neighborhood, religious practices, discrimination, and food security.

Study contacts

Contact information is provided by the study sponsor or research team.

Kayleigh Worek, MS

CONTACT

[email protected]

781-301-6060 ext. 2900

Sponsors and collaborators

Lead sponsor

FSHD Society

Other

Registry information

Important dates

Study start
2024
Primary completion
2060
Study completion
2060
First posted
Feb 13, 2026
Registry last updated
Feb 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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