Overview

This trial is active, not recruiting.

Condition heart, respiratory, disease, functional
Treatment no intervention
Sponsor Centre d'Investigation Clinique et Technologique 805
Start date July 2016
End date September 2016
Trial size 170 participants
Trial identifier NCT02501083, CRHF-MD

Summary

Muscular dystrophies are inherited disorders that affect skeletal muscle. Cardiac and respiratory function may be affected in this group of diseases. The investigators sought to analyze the long term cardiac and respiratory function in patients with muscular dystrophies recquiring home mechnaical ventilation .

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model case-only
Time perspective retrospective

Primary Outcomes

Measure
long term survival
time frame: 10 years

Secondary Outcomes

Measure
hospitalisation for cardiac events
time frame: 10 years
hospitalisation for respiratory events
time frame: 10 years
hospitalisation for other causes
time frame: 10 years
cumulative hospitalisation for cardiac events
time frame: 10 years
cumulative hospitalisation for respiratory events
time frame: 10 years
annual rate of left ventricular ejection fraction decline
time frame: 10 years
annual rate of respiratory vital capacity decline
time frame: 10 years
annual rate of maximal inspiratory pressure decline
time frame: 10 years
annual rate of maximal expiratory pressure decline
time frame: 10 years

Eligibility Criteria

Male or female participants from 18 years up to 70 years old.

Inclusion Criteria: - Age higher or equal to 18 years - Duchenne muscular dystrophy - Becker muscular dystrophy - Sarcoglycanopathy - limb girdle muscular dystrophy - Pompe disease - Steinert disease - FSH disease - Others myopathies Exclusion Criteria: - Minor patient - Sepsis condition

Additional Information

Official title Natural History of Cardiac and Respiratory Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
Principal investigator Abdallah FAYSSOIL, MDPhD
Description Muscular dystrophies are neuromuscular disorders with disability and can affect respiratory muscle function and left ventricular function. Medical management of patients with cardiomyopathy relies mainly on angiotensin-converting-enzyme inhibitors and beta blockers. Respiratory management relies on home mechanical ventilation and couph assist. The investigators aim - to analyze cardiac and respiratory function over time in patients with muscular dystrophies followed at the home mechanical ventilation unit. - to determine the long term impact of the home mechanical ventilation on cardiac and respiratory events and to define the predictive factors associated. Patients will be included retrospectively. Tissue Doppler imaging and Tricuspid annular plane systolic excursion were used for the assessment of the right ventricle. Left ventricular ejection fraction was used for the analysis of the left ventricle. Respiratory function was assessed using the vital capacity, the maximal inspiratory pressure, the maximal expiratory pressure, the peak couph flow rate.
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Centre d'Investigation Clinique et Technologique 805.