Duchenne Muscular Dystrophy Heart Study | DuchenneXchange

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Duchenne Muscular Dystrophy Heart Study

key information

study id #: NCT03443115

condition: Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy

status: recruiting

purpose:

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.

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT03443115

last updated: November 22, 2018

study details

start date: June 27, 2017

estimated completion: February 16, 2018

size / enrollment: 700

primary outcomes:

  • All-cause mortality [ Time Frame: Follow-up completed in March 2020 ]

secondary outcomes:

  • Hospitalization for heart failure [Time Frame: Follow-up completed in March 2020]
  • Dilated cardiomyopathy [Time Frame: Follow-up completed in March 2020]
  • Hospitalization for acute respiratory failure [Time Frame: Follow-up completed in March 2020]

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

exclusion criteria:
Patient refusal to participate to the study

study contacts

sponsor: Hôpital Cochin

contacts: Karim Wahbi (MD, PhD), +33158411653, karim.wahbi@aphp.fr

locations: France