NCT03443115

Duchenne Muscular Dystrophy Heart Study

Multicenter Cohort Study on Duchenne Muscular Dystrophy Cardiomyopathy

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 700 (estimated)
• Sponsor: Hôpital Cochin · Academic / Other
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy
• Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy

Timeline

• Start date: 2017-06-27
• Primary completion: 2018-02-16
• Completion: 2020-03-15
• First posted: 2018-02-22
• Last updated: 2018-02-22

Locations

1 site across 1 country: France