Registry for patients with Facioscapulohumeral Muscular Dystrophy in the UK
The UK Facioscapulohumeral Muscular Dystrophy Patient Registry
This study is creating a registry to collect information from people with Facioscapulohumeral Muscular Dystrophy in the UK to better understand the condition and find future trial participants.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1018 (estimated) |
| Sex | All |
| Sponsor | Newcastle University Academic / other |
| Locations | 1 site (Newcastle upon Tyne) |
| Trial ID | NCT04001582 on ClinicalTrials.gov |
What this trial studies
The UK FSHD Patient Registry is an observational initiative aimed at collecting data from individuals diagnosed with Facioscapulohumeral Muscular Dystrophy (FSHD) across the United Kingdom. Participants can join the registry through referrals or self-discovery and are encouraged to provide annual updates on their condition. The registry aims to enhance understanding of FSHD and facilitate the identification of potential candidates for future clinical trials. It is sponsored by Muscular Dystrophy UK and allows for both self-reporting and optional input from healthcare providers.
Who should consider this trial
Good fit: Ideal candidates for this registry are individuals with a confirmed diagnosis of FSHD residing in the UK.
Not a fit: Patients with confirmed neuromuscular diseases other than FSHD or those living outside of the UK may not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could lead to improved understanding and management of FSHD, potentially accelerating the development of new treatments.
How similar studies have performed: Other patient registries for neuromuscular diseases have shown success in advancing research and treatment options, indicating that this approach is promising.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: \- All patients with a confirmed FSHD diagnosis (or pending diagnosis) who reside in the UK are eligible for inclusion. Exclusion Criteria: * Any confirmed NMD other than FSHD * Living outside of the UK
Where this trial is running
Newcastle upon Tyne
- John Walton Muscular Dystrophy Research Centre — Newcastle upon Tyne, United Kingdom (Recruiting)
Study contacts
- Principal investigator: Chiara Marini-Bettolo, MD, PhD — John Walton Muscular Dystrophy Research Centre
- Study coordinator: Registry Project Manager and Curator
- Email: helen.walker2@newcastle.ac.uk
- Phone: 0191 2418640
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.