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Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

Sponsor
Children's National Research Institute (Other)
Overall Status
Recruiting
CT.gov ID
NCT02921321
Collaborator
(none)
100
Enrollment
1
Location
131
Duration (Months)
0.8
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase

    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.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Pilot Study of Cardiac Magnetic Resonance in Patients With Muscular Dystrophy
    Study Start Date :
    Jan 1, 2014
    Anticipated Primary Completion Date :
    Dec 1, 2024
    Anticipated Study Completion Date :
    Dec 1, 2024

    Outcome Measures

    Primary Outcome Measures

    1. 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 Outcome Measures

    1. Measure the amount of intramyocardial fibrosis using extracellular volume measurements [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. [At the end of the study, up to 10 years]

    3. 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.]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Children's National Health System Washington District of Columbia United States 20010

    Sponsors and Collaborators

    • Children's National Research Institute

    Investigators

    • Principal Investigator: Christopher Spurney, MD, Children's National Research Institute

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    Responsible Party:
    Christopher Spurney, MD, Children's National Research Institute
    ClinicalTrials.gov Identifier:
    NCT02921321
    Other Study ID Numbers:
    • 4193
    • Internal Bridge Funding
    First Posted:
    Oct 3, 2016
    Last Update Posted:
    Mar 2, 2022
    Last Verified:
    Mar 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Keywords provided by Christopher Spurney, MD, Children's National Research Institute
    MRI
    Muscular Dystrophy
    Cardiac
    Late gadolinium enhancement
    Additional relevant MeSH terms:
    Muscular Dystrophies
    Musculoskeletal Diseases
    Genetic Diseases, Inborn

    Study Results

    No Results Posted as of Mar 2, 2022