FSHNCT07409142
BetterLife FSHD: A Patient-driven Health and Research Platform
An observational study of FSH and FSH Muscular Dystrophy, run by FSHD Society at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- FSH, FSH Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy
- What is being tested?
- Observational
- How many people
- 5,000 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Aug 2024to Jan 2060
- How long will I take part?
- 100 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: People with FSHD living in the United States or its territories age one year or older.
Who may be able to join
- 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
Who cannot join
- Individuals residing outside the United States or its territories
- Unable or unwilling to provide consent, or assent, when applicable
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
FSHD Society
Randolph, Massachusetts, 02368
Kayleigh Worek, Program Manager, MS, 781-301-6060 ext. 2900, betterlife@fshdsociety.org
About this study
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
Read the 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.
- Conditions
- FSH, FSH Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1, FSHD, FSHD2
- Being tested
- Observational
- Keywords
- FSHD, Registry, FSH Muscular Dystrophy, FSHD1, FSHD2, Facioscapulohumeral dystrophy, facioscapulohumeral muscular dystrophy
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral, Facioscapulohumeral Muscular Dystrophy 1B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ObservationalOther
Observational study
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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.
Measured: Assessed every 6 months, until study completion (10 years)
Secondary outcomes (14)
Anxiety Patient Reported Outcome Measure
Assesses frequency and impact of anxiety symptoms using a 5-point rating system. Higher scores indicate more anxiety.
Measured: Quarterly, until study completion (10 years)
Depression Patient Reported Outcome Measure
Assesses frequency and impact of depression symptoms using a 5-point rating system. Higher scores indicate more depression.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Yearly until study completion (10 years)
Diagnostic Journey
Survey questions to describe participant experience and barriers getting diagnosed with FSHD.
Measured: One time at Baseline
Healthcare Experiences
Survey questions to describe healthcare utilization and experiences navigating healthcare settings.
Measured: Yearly, until study completion (10 years)
Women's Health
Survey questions about history of menstruation and pregnancy.
Measured: 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.
Measured: Yearly, until study completion (10 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2026
- Study start2 Aug 2024
- Primary completionJan 2060 (estimated)
- Study completionJan 2060 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FSHStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 5,000 participants (estimated)
- Target duration
- 100 Years
Sponsor and investigators
- Lead sponsor
- FSHD Society
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amanda Hill, FSHD Society
- Investigators
- Amanda Hill, MBA, Principal investigator, FSHD Society
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07409142
- Sponsor's protocol ID
- Pro00079388
- Status last verified
- Feb 2026
- First submitted
- 24 Mar 2025
- Last updated
- 20 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- BetterLife FSHD: A Patient-driven Health and Research Platform
- ClinicalTrials.gov ID
- NCT07409142
- Acronym / sponsor ID
- Pro00079388
- Conditions
- FSH, FSH Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1, FSHD, FSHD2
- Keywords
- FSHD, Registry, FSH Muscular Dystrophy, FSHD1, FSHD2, Facioscapulohumeral dystrophy, facioscapulohumeral muscular dystrophy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5,000 participants (estimated)
- Lead sponsor
- FSHD SocietyUniversity, hospital or other
- Study start
- 2 Aug 2024 (actual)
- Primary completion
- Jan 2060 (estimated)
- Study completion
- Jan 2060 (estimated)
- First posted
- 13 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 100 Years
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- People with FSHD living in the United States or its territories age one year or older.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (2 listed)
- Individuals residing outside the United States or its territories
- Unable or unwilling to provide consent, or assent, when applicable
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ObservationalObservational study | Other | – |
Primary outcome
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).
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.
14 secondary outcomes
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)
Women's Health
Time frame: Yearly, until study completion (10 years)
Social Determinants of Health
Time frame: Yearly, until study completion (10 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2026.
- Learn more and enroll in BetterLife FSHDLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Amanda Hill, MBA principal investigator
FSHD Society
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of FSH and FSH Muscular Dystrophy, run by FSHD Society at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kayleigh Worek, MS (781-301-6060 ext. 2900, betterlife@fshdsociety.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Longitudinal Health Data (time frame: Assessed annually from enrollment until study completion (10 years)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2026.