The United Kingdom National Registry for Myotonic Dystrophy

ConditionMyotonic Dystrophy

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeNot listed

SponsorNewcastle University

About this trial

Myotonic dystrophy (dystrophia myotonica - DM) exists in two forms, usually referred to as DM1 (type 1) and DM2 (type 2). Both conditions are genetic disorders but each affects a different gene. DM1 is the most common adult-onset muscular dystrophy, and is thought to affect at least 1 in 8,000 people worldwide.

The aim is to facilitate a questionnaire based research study in order to better characterise and understand the disease in the UK. By maintaining a national registry this will help identify potential participants eligible for clinical trials in the future.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

There are no exclusion criteria for the registry

Trial population

Participants with Myotonic Dystrophy will volunteer to participate in this study. The study will be advertised through neuromuscular disease clinics, the registry website, patient organisations and conferences and meetings throughout the UK.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Patient Registry
Other intervention
Participants who have volunteered to participate will complete various questionnaires relating to their condition.

Treatment groups

900 Participants

are divided into 1 treatment group

Group A: Participants with Myotonic Dystrophy1 intervention

Trial outcomes

Primary outcomes

Patient questionnaire

Patient reported myotonic dystrophy clinical diagnosis, symptoms relating to muscle weakness, motor function, medication use, family history and ethnicity.

Time frame

12 months

Clinician questionnaire

Clinician reported patient cardiac measures, medication use, respiratory measures and genetic confirmation of myotonic dystrophy.

Time frame

12 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Newcastle University

Lead sponsor

Source ClinicalTrials.gov

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