Heart Imaging in Children With Muscular Dystrophy

Terminated

• Conditions: Muscular Dystrophy

• Registration Number: NCT01633242
• Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Brief Summary

Background:

- Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function. However, better types of heart imaging studies are needed to improve treatment of heart problems related to muscular dystrophy. Better heart imaging methods are especially needed for children with muscular dystrophy. Researchers want to test different heart imaging methods in children with muscular dystrophy. They will look at cardiac magnetic resonance imaging (MRI) and standard heart function tests.

Objectives:

- To develop and test new methods for imaging the heart in children with muscular dystrophy.

Eligibility:

- Children and adolescents between 8 and 17 years of age who have muscular dystrophy.

Design:

* Participants will be screened with a physical exam and medical history.

* Participants will provide a blood sample at the start of the study. They will also have heart function tests before having the imaging study.

* Participants will have a cardiac MRI scan that will last up to 60 minutes. Some tests will require a MRI contrast agent (a drug that helps the image appear more clearly on the scan).

Detailed Description

Cardiomyopathy causes significant morbidity and mortality in multiple forms of muscular dystrophy affecting children, including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and subtypes of autosomal recessive limb-girdle muscular dystrophy (LGMD2). Pharmaceutical treatments for the cardiomyopathy of muscular dystrophy, including angiotensin-converting enzyme (ACE) inhibition and beta-adrenergic receptor blockade, afford significant benefit and demonstrate cardiac remodeling in clinical studies. Further studies are needed to identify and characterize more sensitive indicators of cardiac dysfunction in muscular dystrophy subjects to better stratify subjects for entry into clinical protocols. Using the framework of the Cooperative International Neuromuscular Research Group (CINRG) and the Clinical and Translational Science Award (CTSA) consortium, this pilot study will assess cardiac outcome measures in children obtained by gadolinium enhanced cardiac Magnetic Resonance Imaging (MRI) and echocardiographic methods that can be reliably implemented across a consortium of clinical sites devoted to the study of pharmaceutical treatments for muscular dystrophy. These cardiac MRI scans will be shared with the PITT1109 research protocol in all subjects that are enrolled in both studies.

Study Timeline

• Study Start: 2012-02-24
• Study First Submitted: 2012-06-30
• Study First Submitted QC: 2012-06-30
• Study First Posted: 2012-07-04
• Status Verified: 2016-10-11
• Study Completion: 2016-10-11
• Last Update Submitted: 2019-09-12
• Last Update Posted: 2019-09-13

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 2

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Evaluate CMR measures in muscular dystrophy patients

Secondary Outcome Measures

Name	Time	Method
Early Detection
Measurement of Disease Progression
Ultimate identification of the response to developing therapies

Trial Locations

Locations (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike; 🇺🇸 Bethesda, Maryland, United States