CompletedNot Applicable

Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)

United Kingdom177 participantsStarted 2023-07-03

Plain-language summary

The goal of this observational study is to learn about swallowing difficulties (dysphagia) in patients living with myotonic dystrophy type 1 (DM1). The main questions it aims to answer are: * whether the size and structure of the muscles involved in swallowing differ to those without the disease * how the size and structure of muscles may associate with swallowing function and swallowing symptoms in this group. Participants will undergo a range of tests including: * Ultrasound (US) assessment of the muscles involved in swallowing * An x-ray swallowing study (known as videofluoroscopy) * Assessment of swallowing symptoms, including questionnaires * Assessments of mobility, activity and breathing * Assessments of quality of life and wellbeing

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Participants with DM1 Inclusion: * ≥18 years of age * genetically confirmed of DM1 * able to eat \& drink at least five sips of liquid by mouth at one time. Exclusion: * any condition or treatment other than DM1 that potentially influences swallowing muscle composition or function (e.g., a history of stroke or throat cancer). * any patients who are pregnant * patients with congenital or childhood DM1 * patients who are not able to eat or drink anything by mouth.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Swallowing muscle (geniohyoid) size

Timeframe: 16 months

Swallowing muscle (geniohyoid) structure

Timeframe: 16 months

Trial details

NCT IDNCT05865483

SponsorUniversity College, London

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2024-08-23

View on ClinicalTrials.gov More Myotonic Dystrophy 1 trials