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

August 16, 2018 updated by: Centre d'Investigation Clinique et Technologique 805

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

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 .

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Anticipated)

350

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Garches, France, 92380
        • Hôpital Raymond Poincaré

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Adult patients with muscular dystrophies

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
predictive factors for long term survival
Time Frame: 10 years
10 years

Secondary Outcome Measures

Outcome Measure
Time Frame
predictuve factors for cardiac events
Time Frame: 10 years
10 years
predictive factors for respiratory events
Time Frame: 10 years
10 years
cumulative cardiac events incidence
Time Frame: 10 years
10 years
cumulative respiratory events incidence
Time Frame: 10 years
10 years
annual rate of left ventricular ejection fraction decline
Time Frame: 10 years
10 years
annual rate of respiratory vital capacity decline
Time Frame: 10 years
10 years
annual rate of maximal inspiratory pressure decline
Time Frame: 10 years
10 years
annual rate of maximal expiratory pressure decline
Time Frame: 10 years
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Centre d'Investigation Clinique et Technologique 805

Investigators

  • Principal Investigator: Abdallah FAYSSOIL, MDPhD, Hôpital Raymond Poincaré

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2016

Primary Completion (Anticipated)

November 1, 2018

Study Completion (Anticipated)

November 1, 2018

Study Registration Dates

First Submitted

June 26, 2015

First Submitted That Met QC Criteria

July 15, 2015

First Posted (Estimate)

July 17, 2015

Study Record Updates

Last Update Posted (Actual)

August 17, 2018

Last Update Submitted That Met QC Criteria

August 16, 2018

Last Verified

July 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CRHF-MD

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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