CompletedNot Applicable

Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1

France34 participantsStarted 2020-07-03

Plain-language summary

Myotonic dystrophy type 1 (DM1) is one of the most common neuromuscular diseases in adults. As respiratory dysfunction is the most common cause of death in patients with DM1, a respiratory disease progression must be monitored combining symptom screening and respiratory function testing, in order to identify the appropriate time to initiate non invasive ventilation (NIV). Dyspnea, one of the main respiratory symptoms, has been little studied in patients with DM1. The main objective of this study is to provide the first multidimensional description of dyspnea in patients with DM1. The secondary objectives are: * To compare respiratory symptoms according to the presence or not of criteria from respiratory function testing to initiate NIV * To assess associations between dyspnea and respiratory function testing * To assess associations between dyspnea and number of Cytosine Thymine Guanine (CTG) repeats * To assess associations between dyspnea and muscular strength * To assess associations between dyspnea and BMI * To assess associations between dyspnea and anxiety or depression * To assess associations between dyspnea and cognitive impairment * To assess associations between dyspnea and quality of life.

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.

Inclusion Criteria: * patient with myotonic dystrophy type 1 confirmed by genetic analysis * with an age older than 18 years Exclusion Criteria: * an ongoing or recent (i.e. within the last 4 weeks prior to study recruitment) medical condition, including pulmonary exacerbations * patient already under non-invasive mechanical ventilation

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

Dyspnea

Timeframe: Month 6

Trial details

NCT IDNCT04835298

SponsorCHU de Reims

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2021-07-20

View on ClinicalTrials.gov More Myotonic Dystrophy Type 1 (DM1) trials