National registry for myotonic 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

This registry aims to collect and maintain data on individuals diagnosed with myotonic dystrophy, including both type 1 and type 2. Participants can join through referrals from healthcare professionals or by their own initiative, and they will provide information about their condition through a questionnaire. The registry will facilitate ongoing updates to participant information and aims to support future clinical trials by identifying eligible participants. It is sponsored by Muscular Dystrophy UK, Cure-DM, and the Myotonic Dystrophy Support Group.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* All patients with a confirmed Myotonic Dystrophy diagnosis (or pending diagnosis) are eligible for inclusion. Diagnosis will be confirmed via genetic testing results

Exclusion Criteria:

* There are no exclusion criteria for the registry

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 Curator
• Email: registries@ncl.ac.uk
• Phone: 0191 2418640

Similar studies

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

• Understanding long-term progression in myotonic dystrophy type 1 (DM1)

  This study will follow adults with DM1 for at least four years to see how walking, hand function, muscle strength, heart rhythms, and lung function change over time.

  Recruiting Virginia Commonwealth University · Richmond, Virginia · NCT07700225

• Zeleciment Basivarsen (DYNE-101) for people with myotonic dystrophy type 1

  This trial will test whether Zeleciment Basivarsen (DYNE-101) improves symptoms and is safe and tolerable for adults with myotonic dystrophy type 1.

  Recruiting at 44 sites Phase 3 Dyne Therapeutics · La Jolla, California and 43 other locations · NCT07486934

• Quantitative MRI measurement of muscle changes in myotonic dystrophy

  This project will test whether advanced quantitative MRI scans can measure muscle structure and link to disease severity and RNA changes in adults with myotonic dystrophy.

  Recruiting Wake Forest University Health Sciences · Winston-Salem, North Carolina · NCT07362875

• Portable markerless video system to measure walking in adults with neuromuscular conditions or healthy volunteers.

  This project will test a portable video device that measures walking patterns to see if it can track gait changes in adults with neuromuscular diseases and in healthy volunteers.

  Recruiting Institut de Myologie, France · Paris · NCT07321977

• SAR446268 gene therapy for non-congenital myotonic dystrophy type 1

  This will try a single intravenous dose of SAR446268 gene therapy to lower toxic DMPK mRNA and improve muscle function in people aged 10 to 55 with non‑congenital myotonic dystrophy type 1.

  Recruiting at 10 sites Phase 1/2 Sanofi · Gainesville, Florida and 9 other locations · NCT06844214

• Assessing Readiness and Endpoints for Pediatric Myotonic Dystrophy

  This study is trying to improve how we understand and measure the effects of congenital and childhood myotonic dystrophy to help create better treatments for kids with these conditions.

  Recruiting Virginia Commonwealth University · Little Rock, Arkansas and 3 other locations · NCT06747884

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.