Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1

Myotonic Dystrophy Type 1 (DM1) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04835298
• Other study ID #: PO20065*
• Status: Completed
• Start date: July 3, 2020
• Est. completion date: June 14, 2022

Study information

• Verified date: June 2022
• Source: CHU de Reims
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: June 14, 2022
• Est. primary completion date: July 20, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy Type 1 (DM1)

Intervention

Other:
• Dyspnea
questionnaires

Locations

Country	Name	City	State
France	Chu Reims	Reims

Sponsors (1)

Lead Sponsor	Collaborator
CHU de Reims

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Dyspnea	Borg scale at rest and after 6 minute walking test	Month 6