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Clinical Trials/NCT02501083
NCT02501083
Unknown
Not Applicable

Natural History of Cardiac and Respiratory Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation

Centre d'Investigation Clinique et Technologique 8051 site in 1 country350 target enrollmentJuly 2016

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Heart, Respiratory, Disease, Functional
Sponsor
Centre d'Investigation Clinique et Technologique 805
Enrollment
350
Locations
1
Primary Endpoint
predictive factors for long term survival
Last Updated
7 years ago

Overview

Brief 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 .

Detailed 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.

Registry
clinicaltrials.gov
Start Date
July 2016
End Date
November 2018
Last Updated
7 years ago
Study Type
Observational
Sex
All

Investigators

Sponsor
Centre d'Investigation Clinique et Technologique 805
Responsible Party
Sponsor

Eligibility Criteria

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

Outcomes

Primary Outcomes

predictive factors for long term survival

Time Frame: 10 years

Secondary Outcomes

  • predictuve factors for cardiac events(10 years)
  • predictive factors for respiratory events(10 years)
  • cumulative cardiac events incidence(10 years)
  • cumulative respiratory events incidence(10 years)
  • annual rate of maximal inspiratory pressure decline(10 years)
  • annual rate of maximal expiratory pressure decline(10 years)
  • annual rate of left ventricular ejection fraction decline(10 years)
  • annual rate of respiratory vital capacity decline(10 years)

Study Sites (1)

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