National registry for myotonic dystrophy in the UK
The UK National Registry for Myotonic Dystrophy
This study is setting up a registry to gather information from people with myotonic dystrophy to help improve future treatments and find participants for clinical trials.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 900 (estimated) |
| Sex | All |
| Sponsor | Newcastle University Academic / other |
| Locations | 1 site (Newcastle upon Tyne) |
| Trial ID | NCT04003363 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this registry are individuals with a confirmed diagnosis of myotonic dystrophy.
Not a fit: Patients without a diagnosis of myotonic dystrophy or those who do not wish to participate in self-reporting may not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could enhance understanding of myotonic dystrophy and improve patient access to clinical trials.
How similar studies have performed: Other registries for genetic disorders have shown success in improving patient outcomes and facilitating research, indicating a positive precedent for this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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 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.