Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1

June 14, 2022 updated by: CHU de Reims

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

34

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Reims, France, 51092
        • CHU Reims

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

adult patients with myotonic dystrophy type 1

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Myotonic dystrophy type 1
adult patients with myotonic dystrophy type 1
Other: Dyspnea
questionnaires

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Dyspnea
Time Frame: Month 6
Borg scale at rest and after 6 minute walking test
Month 6

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CHU de Reims

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 3, 2020

Primary Completion (Actual)

July 20, 2021

Study Completion (Actual)

June 14, 2022

Study Registration Dates

First Submitted

March 31, 2021

First Submitted That Met QC Criteria

April 6, 2021

First Posted (Actual)

April 8, 2021

Study Record Updates

Last Update Posted (Actual)

June 15, 2022

Last Update Submitted That Met QC Criteria

June 14, 2022

Last Verified

June 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PO20065*

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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