Cardiac Involvement in Patients with Duchenne/Becker Muscular Dystrophy
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 59
- Locations
- 1
- Primary Endpoint
- Left ventricular ejection fraction
Study Overview
Brief Summary
This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging.
Detailed Description
Muscular dystrophy leads to progressive loss of function in all muscles during childhood and adolescence, including the heart. The usual method to evaluate the heart is echocardiography, emphasizing few parameters. Cardiac magnetic resonance imaging is not as widely available as echocardiography, but early changes can be detected before they become visible on echocardiography. In this study, the investigators compare the methods of measuring heart function in order to find the best measurements for follow up and to see how fast the degenerative changes occur in the hearts of patients with muscular dystrophy.
Study Design
- Study Type
- Observational
- Observational Model
- Case Only
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 8 Years to 18 Years (Child, Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •Boys aged 8 to 18 years with DMD/BMD confirmed genetically or by muscle biopsy
- •Informed consent
Exclusion Criteria
- •Other clinically significant concomitant disease states (e.g., renal failure)
- •Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia, etc. of the participant or his/her parents or legal caregivers,
- •Inability to lie still for the duration of the imaging procedures (approximately 45 minutes each for echocardiography and CMR)
- •MR-incompatible implanted or accidentally incorporated metal device or claustrophobia that prohibits use of magnetic resonance imaging
Outcomes
Primary Outcomes
Left ventricular ejection fraction
Time Frame: 3 years per patient
Secondary Outcomes
- NT-proBNP(3 years per patient)
- Quantification of fibrosis by LGE/T1 mapping(3 years per patient)