Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

September 19, 2023 updated by: Christopher Spurney, Children's National Research Institute

Pilot Study of Cardiac Magnetic Resonance 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.

Study Overview

Status

Active, not recruiting

Conditions

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 Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Health System

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Male or females at least 6 years of age with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
At the end of each MRI scan through study completion, up to 5 years.

Secondary Outcome Measures

Outcome Measure
Time Frame
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.
At the end of each MRI scan through study completion, up to 5 years.
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
At the end of the study, up to 10 years
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.
At the end of each MRI scan through study completion, up to 5 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's National Research Institute

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2014

Primary Completion (Estimated)

December 1, 2024

Study Completion (Estimated)

December 1, 2024

Study Registration Dates

First Submitted

August 29, 2016

First Submitted That Met QC Criteria

September 29, 2016

First Posted (Estimated)

October 3, 2016

Study Record Updates

Last Update Posted (Actual)

September 21, 2023

Last Update Submitted That Met QC Criteria

September 19, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 4193
  • Internal Bridge funding (Other Identifier: Children's Research Institute, Children's National)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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