Trial record 1 of 1 for: NCT03443115

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Duchenne Muscular Dystrophy Heart Study (DMD-HS)

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ClinicalTrials.gov Identifier: NCT03443115

Recruitment Status : Unknown

Verified February 2018 by Karim Wahbi, Hôpital Cochin.
Recruitment status was: Recruiting

First Posted : February 22, 2018

Last Update Posted : February 22, 2018

View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborator:

Association Monégasque contre les Myopathies

Information provided by (Responsible Party):

Karim Wahbi, Hôpital Cochin

Study Description

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.

Condition or disease
Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy

Study Design

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Study Type :	Observational
Estimated Enrollment :	700 participants
Observational Model:	Cohort
Time Perspective:	Retrospective
Official Title:	Multicenter Cohort Study on Duchenne Muscular Dystrophy Cardiomyopathy
Actual Study Start Date :	June 27, 2017
Actual Primary Completion Date :	February 16, 2018
Estimated Study Completion Date :	March 15, 2020

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Familial dilated cardiomyopathy Duchenne and Becker muscular dystrophy X-linked dilated cardiomyopathy

MedlinePlus related topics: Cardiomyopathy Muscular Dystrophy

Genetic and Rare Diseases Information Center resources: Muscular Dystrophy Becker Muscular Dystrophy Duchenne Muscular Dystrophy Dilated Cardiomyopathy

U.S. FDA Resources

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

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 ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	Child, Adult, Older Adult
Sexes Eligible for Study:	Male
Accepts Healthy Volunteers:	No
Sampling Method:	Probability Sample

Study Population

Consecutive patients diagnosed with Duchenne muscular dystrophy and referred to reference centers for neuromuscular diseases for the management of their condiction.

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

Patient refusal to participate to the study

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03443115

Contacts

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Contact: Karim Wahbi, MD, PhD	+33158411653	karim.wahbi@aphp.fr

Locations

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France
Cochin Reference Center for Neuromuscular Diseases	Recruiting
Paris, France, 75014
Contact: Karim Wahbi, MD, PhD +33158411653 karim.wahbi@aphp.fr

Sponsors and Collaborators

Hôpital Cochin

Association Monégasque contre les Myopathies

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Porcher R, Desguerre I, Amthor H, Chabrol B, Audic F, Rivier F, Isapof A, Tiffreau V, Campana-Salort E, Leturcq F, Tuffery-Giraud S, Ben Yaou R, Annane D, Amedro P, Barnerias C, Becane HM, Behin A, Bonnet D, Bassez G, Cossee M, de La Villeon G, Delcourte C, Fayssoil A, Fontaine B, Godart F, Guillaumont S, Jaillette E, Laforet P, Leonard-Louis S, Lofaso F, Mayer M, Morales RJ, Meune C, Orlikowski D, Ovaert C, Prigent H, Saadi M, Sochala M, Tard C, Vaksmann G, Walther-Louvier U, Eymard B, Stojkovic T, Ravaud P, Duboc D, Wahbi K. Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy-analysis of registry data. Eur Heart J. 2021 May 21;42(20):1976-1984. doi: 10.1093/eurheartj/ehab054.

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Responsible Party:	Karim Wahbi, MD, PhD, Hôpital Cochin
ClinicalTrials.gov Identifier:	NCT03443115
Other Study ID Numbers:	DMD-HS
First Posted:	February 22, 2018 Key Record Dates
Last Update Posted:	February 22, 2018
Last Verified:	February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	No

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Studies a U.S. FDA-regulated Drug Product:	No
Studies a U.S. FDA-regulated Device Product:	No

Additional relevant MeSH terms:

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Muscular Dystrophies Muscular Dystrophy, Duchenne Cardiomyopathies Cardiomyopathy, Dilated Heart Diseases Cardiovascular Diseases Muscular Disorders, Atrophic Muscular Diseases	Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Cardiomegaly Laminopathies

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