- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03443115
Duchenne Muscular Dystrophy Heart Study (DMD-HS)
Multicenter Cohort Study on Duchenne Muscular Dystrophy Cardiomyopathy
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.
Study Overview
Status
Study Type
Enrollment (Anticipated)
Contacts and Locations
Study Contact
- Name: Karim Wahbi, MD, PhD
- Phone Number: +33158411653
- Email: karim.wahbi@aphp.fr
Study Locations
-
-
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Paris, France, 75014
- Recruiting
- Cochin Reference Center for Neuromuscular Diseases
-
Contact:
- Karim Wahbi, MD, PhD
- Phone Number: +33158411653
- Email: karim.wahbi@aphp.fr
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
Inclusion Criteria:
- Genetically-proven Duchenne Muscular Dystrophy (DMD mutation)
- Covering by social security
Exclusion Criteria:
- Patient refusal to participate to the study
Study Plan
How is the study designed?
Design Details
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
All-cause mortality
Time Frame: Follow-up completed in March 2020
|
Follow-up completed in March 2020
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Hospitalization for heart failure
Time Frame: Follow-up completed in March 2020
|
Follow-up completed in March 2020
|
Dilated cardiomyopathy
Time Frame: Follow-up completed in March 2020
|
Follow-up completed in March 2020
|
Hospitalization for acute respiratory failure
Time Frame: Follow-up completed in March 2020
|
Follow-up completed in March 2020
|
Collaborators and Investigators
Sponsor
Collaborators
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Heart Diseases
- Cardiovascular Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Muscular Disorders, Atrophic
- Cardiomegaly
- Laminopathies
- Muscular Dystrophies
- Cardiomyopathies
- Cardiomyopathy, Dilated
- Muscular Dystrophy, Duchenne
Other Study ID Numbers
- DMD-HS
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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