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NCT Number: NCT02921321

Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

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.

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Key information

Conditions

Muscular Dystrophies Cardiac Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Muscular Diseases Muscular Disorders, Atrophic Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's National Health System

Washington D.C., District of Columbia, 20010, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Myocardial characterization of fibrosis in patients with muscular dystrophy using contrast based magnetic resonance imaging

    Time frame: At the end of each MRI scan through study completion, up to 5 years.

Secondary outcomes

  1. Measure the amount of intramyocardial fibrosis using extracellular volume measurements

    Time frame: At the end of each MRI scan through study completion, up to 5 years.

  2. Identification and validation of serum biomarker ST2 (Interleukin 1 receptor-like 1 protein ) in the presence of myocardial fibrosis.

    Time frame: At the end of the study, up to 10 years

  3. Measure regional myocardial strain and correlate with presence of myocardial fibrosis

    Time frame: At the end of each MRI scan through study completion, up to 5 years.

Sponsors and collaborators

Lead sponsor

Children's National Research Institute

Other

Registry information

Official study title

Pilot Study of Cardiac Magnetic Resonance in Patients With Muscular Dystrophy

Important dates

Study start
2014
Primary completion
2024
Study completion
2024
First posted
Oct 3, 2016
Registry last updated
Dec 11, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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