Pilot Study of Cardiac MR in Patients With Muscular Dystrophy
NCT ID: NCT02921321
Last Updated: 2024-12-11
Study Results
Results pending
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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Recruitment Status
COMPLETED
Total Enrollment
60 participants
Study Classification
OBSERVATIONAL
Study Start Date
2014-01-31
Study Completion Date
2024-12-09
Brief Summary
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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.
Detailed Description
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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.
Conditions
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Muscular Dystrophies
Cardiac Fibrosis
Genetic Diseases, Inborn
Musculoskeletal Diseases
Keywords
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MRI
Muscular Dystrophy
Cardiac
Late gadolinium enhancement
Study Design
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Observational Model Type
COHORT
Study Time Perspective
PROSPECTIVE
Eligibility Criteria
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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
* 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
Minimum Eligible Age
6 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
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Children's National Research Institute
OTHER
Sponsor Role
lead
Responsible Party
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Christopher Spurney
MD
Responsibility Role
PRINCIPAL_INVESTIGATOR
Principal Investigators
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Christopher Spurney, MD
Role: PRINCIPAL_INVESTIGATOR
Children's National Research Institute
Locations
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Children's National Health System
Washington D.C., District of Columbia, United States
Site Status
Countries
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United States
References
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Other Identifiers
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Internal Bridge Funding
Identifier Type: OTHER
Identifier Source: secondary_id
4193
Identifier Type: -
Identifier Source: org_study_id