Duchenne Muscular Dystrophy Heart Study

Multicenter Cohort Study on Duchenne Muscular Dystrophy Cardiomyopathy

Status

UNKNOWN

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03443115

Acronym

DMD-HS

Enrollment

700

Registered

2018-02-22

Start date

2017-06-27

Completion date

2020-03-15

Last updated

2018-02-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

MALE

Healthy volunteers

Inclusion criteria

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

Exclusion criteria

* Patient refusal to participate to the study

Design outcomes

Primary

Measure	Time frame
All-cause mortality	Follow-up completed in March 2020

Secondary

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

Countries

France

Contacts

Primary ContactKarim Wahbi, MD, PhD

karim.wahbi@aphp.fr+33158411653

Outcome results