Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1

NCT ID: NCT04835298

Last Updated: 2022-06-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

34 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-07-03

Study Completion Date

2022-06-14

Brief Summary

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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.

Detailed Description

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Conditions

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Myotonic Dystrophy Type 1 (DM1)

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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Myotonic dystrophy type 1

adult patients with myotonic dystrophy type 1

Dyspnea

Intervention Type OTHER

questionnaires

Interventions

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Dyspnea

questionnaires

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CHU de Reims

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Chu Reims

Reims, , France

Site Status

Countries

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France

Other Identifiers

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PO20065*

Identifier Type: -

Identifier Source: org_study_id

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