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Duchenne Muscular Dystrophy Heart Study (DMD-HS)

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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

Tracking Information
First Submitted Date February 16, 2018
First Posted Date February 22, 2018
Last Update Posted Date February 22, 2018
Actual Study Start Date June 27, 2017
Actual Primary Completion Date February 16, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: February 16, 2018)
All-cause mortality [ Time Frame: Follow-up completed in March 2020 ]
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures
 (submitted: February 16, 2018)
  • 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 ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Duchenne Muscular Dystrophy Heart Study
Official Title Multicenter Cohort Study on Duchenne Muscular Dystrophy 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.

Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Retrospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition
  • Duchenne Muscular Dystrophy
  • Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: February 16, 2018)
700
Original Estimated Enrollment Same as current
Estimated Study Completion Date March 15, 2020
Actual Primary Completion Date February 16, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • Patient refusal to participate to the study
Sex/Gender
Sexes Eligible for Study: Male
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Karim Wahbi, MD, PhD +33158411653 karim.wahbi@aphp.fr
Listed Location Countries France
Removed Location Countries  
 
Administrative Information
NCT Number NCT03443115
Other Study ID Numbers DMD-HS
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Karim Wahbi, Hôpital Cochin
Study Sponsor Hôpital Cochin
Collaborators Association Monégasque contre les Myopathies
Investigators Not Provided
PRS Account Hôpital Cochin
Verification Date February 2018