At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Patient Registry (other)
- How long the study runs
- Study runs about 320 months (dates as stated)
- About the drug or intervention
- Patient Registry — other: Participants who have volunteered to participate will complete various questionnaires relating to their condition.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Observing health over time
- Ages
- Not specified
- Who
- All
- Number of participants
- 1,018
- Started
- 2013-05
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for facioscapulohumeral muscular dystrophy
- • Clinical study - 1,018 participants
- • Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000
Who can take part?
- • Adults
- • Diagnosed with facioscapulohumeral muscular dystrophy
Where?
- • Newcastle upon Tyne - John Walton Muscular Dystrophy Research Centre
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000. FSHD is an autosomal dominant genetic disease and is estimated to affect up to 3,000 people in the UK. The patient registry facilitates a questionnaire based research study to better characterise and understand the disease in the UK, and helps to identify potential participants eligible for clinical trials.
More detail
The UK FSHD Patient Registry (https://www.fshd-registry.org/uk/) recruits any individual, from anywhere within the United Kingdom, with a diagnosis of FSHD. The registry is sponsored by Muscular Dystrophy UK. Participants may be referred to the registry by health care professionals, genetic testing/laboratory centres who are aware of the registry etc. Alternatively, a participant may have discovered the registry via promotional activities or by their own online searches. After completing the consent process, participants are able to enter information on to the registry platform (note all forms are available to view on the registry website before joining the registry). This is an ongoing database and all participants are invited to update their information on an annual basis. The database is designed to be self reporting, however where specialised clinical or genetic information is required, the neuromuscular specialist in charge of the participants care can be invited to provide some additional information. The participant is able to select a health care provider from a pre-populated list at registration stage, if they wish to (optional feature). This information is included in the patient information and consent. Relevant R\&D approval has been recieved.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Not specified
- Who can join: All genders
What the study is looking for
- ✓\- All patients with a confirmed FSHD diagnosis (or pending diagnosis) who reside in the UK are eligible for inclusion.
Who cannot take part
- ✗Any confirmed NMD other than FSHD
- ✗Living outside of the UK
See the full criteria
Where Is This Study? (1 UK site)
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne NE1 3BZ, United Kingdom
How to Get in Touch
Registry Project Manager and Curator
Sponsor contactCONTACT
Registries Team
Sponsor contactCONTACT