Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

Musculoskeletal Diseases Clinical Trial

Official title:

Pilot Study of Cardiac Magnetic Resonance in Patients With Muscular Dystrophy

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02921321
• Other study ID #: 4193
• Secondary ID: Internal Bridge
• Status: Active, not recruiting
• Start date: January 2014
• Est. completion date: December 2024

Study information

• Verified date: September 2023
• Source: Children's National Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Muscular Dystrophy can affect the skeletal muscles and also the heart and breathing muscles, causing significant morbidity and mortality. As patients are now living longer, treatment of muscular dystrophies involves drugs that help improve heart function. However, better types of heart imaging studies are needed to understand how these treatments work. Researchers want to improve heart imaging to identify earlier indicators of heart dysfunction in muscular dystrophy patients and how these are changed by medical treatment. The new imaging indicators will also help identify candidates for entry into future clinical trials.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 100
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

• Diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy

Exclusion Criteria:

• Day-time users of continuous positive airway pressure (CPAP)

• Sip ventilator users

• Invasive ventilator dependent

• Pregnant minors or adults (when uncertain, participants will undergo urine testing) or lactating minors and adults

• Females who are nursing and who do not plan to discard breast milk for 24 hours

• Decompensated congestive heart failure (unable to lie flat during CMR)

• Impaired renal excretory function (calculated Glomerular Filtration Rate less than 30 milliliters/min)

• Contra-indications to Magnetic Resonance Imaging:

• Cardiac pacemaker or implantable defibrillator

• Cerebral aneurysm clip

• Neural stimulator

• Metallic ocular foreign body

• Any implanted device (i.e. insulin pump, drug infusion device)

• Claustrophobia

• Metal shrapnel or bullet

• Investigator assessment of inability to comply with protocol

• Unable/unwilling to lie still throughout the research procedure or who require sedation

• Persons with cognitive impairment

Related Conditions & MeSH terms

• Cardiac Fibrosis
• Genetic Diseases, Inborn
• Muscular Dystrophies
• Musculoskeletal Diseases

Locations

Country	Name	City	State
United States	Children's National Health System	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Children's National Research Institute

Country where clinical trial is conducted

United States, 

References & Publications (51)

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* Note: There are 51 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Myocardial characterization of fibrosis in patients with muscular dystrophy using contrast based magnetic resonance imaging		At the end of each MRI scan through study completion, up to 5 years.
Secondary	Measure the amount of intramyocardial fibrosis using extracellular volume measurements		At the end of each MRI scan through study completion, up to 5 years.
Secondary	Identification and validation of serum biomarker ST2 (Interleukin 1 receptor-like 1 protein ) in the presence of myocardial fibrosis.		At the end of the study, up to 10 years
Secondary	Measure regional myocardial strain and correlate with presence of myocardial fibrosis		At the end of each MRI scan through study completion, up to 5 years.