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

Heart, Respiratory, Disease, Functional Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02501083
• Other study ID #: CRHF-MD
• Status: Active, not recruiting
• Start date: July 2016
• Est. completion date: November 2018

Study information

• Verified date: July 2018
• Source: Centre d'Investigation Clinique et Technologique 805
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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 .

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 350
• Est. completion date: November 2018
• Est. primary completion date: November 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Heart, Respiratory, Disease, Functional
• Muscular Dystrophies

Intervention

Other:
• no intervention
The study will not include any intervention.

Locations

Country	Name	City	State
France	Hopital Raymond Poincare	Garches

Sponsors (1)

Lead Sponsor	Collaborator
Centre d'Investigation Clinique et Technologique 805

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	predictive factors for long term survival		10 years
Secondary	predictuve factors for cardiac events		10 years
Secondary	predictive factors for respiratory events		10 years
Secondary	cumulative cardiac events incidence		10 years
Secondary	cumulative respiratory events incidence		10 years
Secondary	annual rate of left ventricular ejection fraction decline		10 years
Secondary	annual rate of respiratory vital capacity decline		10 years
Secondary	annual rate of maximal inspiratory pressure decline		10 years
Secondary	annual rate of maximal expiratory pressure decline		10 years