Duchenne Muscular Dystrophy Heart Study

• Conditions: Duchenne Muscular Dystrophy; Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy

• Registration Number: NCT03443115
• Lead Sponsor: Hôpital Cochin

Brief Summary

Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020.

Inclusion of the data relative to genetic diagnosis, clinical characteristics at baseline, cardiac and respiratory workup, medical treatments (ACE inhibitors, steroids), surgical procedures, and occurrence during follow-up of cardiac, respiratory and fatal events.

Objectives are to describe long-term natural history of the disease, vital prognosis, genotype-phenotype correlations, effect of treatments.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-06-27
• Status Verified: 2018-02
• Primary Completion: 2018-02-16
• Study First Submitted: 2018-02-16
• Study First Submitted QC: 2018-02-16
• Last Update Submitted: 2018-02-16
• Study First Posted: 2018-02-22
• Last Update Posted: 2018-02-22
• Study Completion: 2020-03-15

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Male
• Target Recruitment: 700

Inclusion Criteria

• Genetically-proven Duchenne Muscular Dystrophy (DMD mutation)
• Covering by social security

Exclusion Criteria

• Patient refusal to participate to the study

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
All-cause mortality	Follow-up completed in March 2020

Secondary Outcome Measures

Name	Time	Method
Hospitalization for acute respiratory failure	Follow-up completed in March 2020
Hospitalization for heart failure	Follow-up completed in March 2020
Dilated cardiomyopathy	Follow-up completed in March 2020

Trial Locations

Locations (1)

Cochin Reference Center for Neuromuscular Diseases; 🇫🇷 Paris, France