Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy

NCT ID: NCT02470962

Last Updated: 2025-03-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

59 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-06-01

Study Completion Date

2022-03-22

Brief Summary

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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

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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.

Conditions

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Duchenne / Becker Muscular Dystrophy

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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Patients with muscular dystrophy

Boys aged 8 to 18 years with muscular dystrophy of the Duchenne / Becker type

No interventions assigned to this group

Children without heart disease

Children without heart disease, aged 8-18 years, as CMR comparison group

Observation

Intervention Type OTHER

Observation by serial echocardiography with extended techniques and cardiac magnetic resonance imaging

Interventions

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Observation

Observation by serial echocardiography with extended techniques and cardiac magnetic resonance imaging

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

8 Years

Maximum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

Yes

Sponsors

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University Children's Hospital, Zurich

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Barbara EU Burkhardt, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Zürich, Switzerland

Locations

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Children's Hospital

Zurich, Canton of Zurich, Switzerland

Site Status

Countries

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Switzerland

Other Identifiers

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DMD-Herz

Identifier Type: -

Identifier Source: org_study_id

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