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Completed

NCT Number: NCT04835298

Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1

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.

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Key information

Conditions

Myotonic Dystrophy Type 1 (DM1) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Chu Reims

Reims, 51092, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

dyspnea

Other

questionnaires

Primary outcomes

  1. Dyspnea

    Time frame: Month 6

    Borg scale at rest and after 6 minute walking test

Sponsors and collaborators

Lead sponsor

CHU de Reims

Other

Registry information

Official study title

Characters) Dyspnea Assessment in Adult Patients With Myotonic Dystrophy Type 1: a Monocentric Pilot Study

Important dates

Study start
2020
Primary completion
2021
Study completion
2022
First posted
Apr 8, 2021
Registry last updated
Jun 15, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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