Registry for patients with Facioscapulohumeral Muscular Dystrophy in the UK

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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: registries@newcastle.ac.uk
• Phone: 0191 2418640

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Apitegromab for FSHD in Adults (Phase 2)

  This study tests whether apitegromab works as a standalone treatment for adults with FSHD.

  Recruiting Phase 2 Scholar Rock, Inc. · Austin, Texas · NCT07435129

• BetterLife FSHD: a patient-driven health 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.

  Recruiting FSHD Society · Randolph, Massachusetts · NCT07409142

• Del-brax (AOC 1020) intravenous treatment for people with FSHD

  This study will try regular IV infusions of del-brax (AOC 1020) in adults with FSHD to see if it slows muscle weakness and helps preserve walking ability.

  Recruiting at 28 sites Phase 3 Avidity Biosciences, Inc. · Orange, California and 45 other locations · NCT07038200

• Personalized exercise treatment for adults with rare neuromuscular disorders

  This study is testing if a personalized exercise program can help adults with rare neuromuscular disorders improve their balance and physical function better than regular care.

  Recruiting at 2 sites Oslo University Hospital · Bergen and 4 other locations · NCT06708468

• Developing new biomarkers for muscular dystrophies

  This study is trying to find easier and less painful ways to measure how serious muscular dystrophies are by looking at urine and blood samples and using simple tests, to help improve diagnosis and treatment for people with these conditions.

  Recruiting at 4 sites Massachusetts General Hospital · Boston, Massachusetts and 4 other locations · NCT05019625

• Registry for Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy

  This study is creating a registry for people with myotonic dystrophy or facioscapulohumeral muscular dystrophy to share their health information and connect with researchers working to learn more about these conditions.

  Recruiting University of Rochester · Rochester, New York · NCT00082108

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.