Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years

• Conditions: Myotonic Dystrophy, Steinert

• Registration Number: NCT03764150
• Lead Sponsor: University Hospital, Lille

Brief Summary

The aim of this study is to determine the factors associated with alveolar hypoventilation in terms of cognitive impairment, daytime sleepiness, respiratory function, nocturnal respiratory events This evaluation will clarify the clinical phenotypes of respiratory disease in myotonic steinert dystrophy.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06-30
• Study First Submitted: 2018-11-19
• Study First Submitted QC: 2018-12-03
• Study First Posted: 2018-12-04
• Status Verified: 2021-01
• Last Update Submitted: 2021-01-06
• Last Update Posted: 2021-01-07
• Primary Completion: 2023-09
• Study Completion: 2023-09

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 160

Inclusion Criteria

• Myotonic dystrophy of Steinert genetically confirmed (number of known CTG repeats), in its classical form of adult

Exclusion Criteria

• Inability to perform EFR assessment or polysomnography
• Moderate to severe asthma according to the GINA classification
• moderate to severe COPD (> stage 1 of the GOLD recommendations)
• Other significant respiratory pathology
• Patient already on NIV, CPAP or under oxygen therapy
• Impossibility of receiving enlightened information
• Inability to participate in the entire study

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change of measurement of spirometry	every year during 5 years	composite predictive factors of alveolar hypoventilation
Change respiratory force	every year during 5 years	composite predictive factors of alveolar hypoventilation to change Inspiratory and expiratory force
Change CO2 response (Read test)	every year during 5 years	composite predictive factors of alveolar hypoventilation
Change subjective sleepiness (Epworth)	every year during 5 years	composite predictive factors of alveolar hypoventilation
change Krupp's Fatigue Severity Scale (KFSS)	every year during 5 years	composite predictive factors of alveolar hypoventilation This is a short questionnaire of 9 questions to which the patient responds on a Likert scale ranging from 1 to 7
change cognitive impairment by Trail Making Test A & B	every year during 5 years	composite predictive factors of alveolar hypoventilation Trail Making Test is a neuropsychological test of visual attention and task switching. It consists of two parts in which the subject is instructed to connect a set of 25 dots as quickly as possible while still maintaining accuracy.
change Oximetry	every year during 5 years	composite predictive factors of alveolar hypoventilation

Trial Locations

Locations (6)

Ap-Hp Hu Paris Site Raymond Poincare; 🇫🇷 Garches, France

Centre Hospitalier Regional D' Angers; 🇫🇷 Angers, France

Hôpital Calmette, CHRU; 🇫🇷 Lille, France

Ap-Hp Hu Pitie Salpetriere; 🇫🇷 Paris, France

Chu Montpellier; 🇫🇷 Montpellier, France

Chu de Nantes :Site Hôtel-Dieu- Hme - Nantes 1; 🇫🇷 Nantes, France