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Status unknownNCT03443115DMD-HSUpdated Feb 22, 2018

Duchenne Muscular Dystrophy Heart Study

An observational study in Duchenne Muscular Dystrophy and Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy, sponsored by Hôpital Cochin. Status unknown at 1 site in France. Open to male participants. Per ClinicalTrials.gov, last updated 2018-02-22.

Sponsored by Hôpital Cochin · Observational

The sponsor has not verified this record recently (last verified Feb 2018), so the status shown — last known as Recruiting — may be out of date.
Study type
Observational
Model
Cohort
Time perspective
Retrospective
Enrollment
700
Sex
Male
01

Study 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.

02

Conditions studied

  • Duchenne Muscular Dystrophy
  • Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
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.

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

  • Patient refusal to participate to the study
04

Study design

Observational model
Cohort
Time perspective
Retrospective
Enrollment
700 participants (estimated)
Patient registry
No
05

What researchers measure

Primary outcomes

  1. All-cause mortality

    Time frame: Follow-up completed in March 2020

Secondary outcomes

  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

06

Study locations

1 of 1 sites recruiting
  • Cochin Reference Center for Neuromuscular Diseases
    Paris, 75014, France
    Recruiting
07

References and documents

Publications

  • 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. PubMed 33748842 ↗

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT03443115
Lead sponsor
Hôpital Cochin
Collaborators
Association Monégasque contre les Myopathies
Responsible party
Karim Wahbi (MD, PhD, Hôpital Cochin) — Principal investigator
First posted
Feb 22, 2018
Start date
Jun 27, 2017
Primary completion
Feb 16, 2018
Completion
Mar 15, 2020 (estimated)
Last update
Feb 22, 2018

Study contacts

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

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is status unknown, as verified in Feb 2018. You cannot join it, but the record below documents what was studied.

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