ClinicalTrials.gov
ClinicalTrials.gov Menu

Duchenne Muscular Dystrophy Heart Study (DMD-HS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03443115
Recruitment Status : Recruiting
First Posted : February 22, 2018
Last Update Posted : February 22, 2018
Sponsor:
Collaborator:
Association Monégasque contre les Myopathies
Information provided by (Responsible Party):
Karim Wahbi, Hôpital Cochin

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 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





Primary Outcome Measures :
  1. All-cause mortality [ Time Frame: Follow-up completed in March 2020 ]

Secondary Outcome Measures :
  1. Hospitalization for heart failure [ Time Frame: Follow-up completed in March 2020 ]
  2. Dilated cardiomyopathy [ Time Frame: Follow-up completed in March 2020 ]
  3. Hospitalization for acute respiratory failure [ Time Frame: Follow-up completed in March 2020 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
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

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

Locations
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

Responsible Party: Karim Wahbi, MD, PhD, Hôpital Cochin
ClinicalTrials.gov Identifier: NCT03443115     History of Changes
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

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

Additional relevant MeSH terms:
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