Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy

Duchenne / Becker Muscular Dystrophy Clinical Trial

Official title:

Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02470962
• Other study ID #: DMD-Herz
• Status: Active, not recruiting
• Start date: May 2015
• Est. completion date: April 2020

Study information

• Verified date: July 2019
• Source: University Children's Hospital, Zurich
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 40
• Est. completion date: April 2020
• Est. primary completion date: April 2020
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Male
• Age group: 8 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne / Becker Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

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

Locations

Country	Name	City	State
Switzerland	Children's Hospital	Zürich	ZH

Sponsors (1)

Lead Sponsor	Collaborator
University Children's Hospital, Zurich

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Left ventricular ejection fraction		3 years per patient
Secondary	Quantification of fibrosis by LGE/T1 mapping		3 years per patient
Secondary	NT-proBNP		3 years per patient