Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

NCT02921321 | Completed

• 2 other identifiers: 4193Internal Bridge Funding
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

MRI; Muscular Dystrophy; Cardiac; Late gadolinium enhancement

Outcome Measures

Primary Outcomes (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 Outcomes (3)

• Measure the amount of intramyocardial fibrosis using extracellular volume measurements

  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.

  At the end of the study, up to 10 years

• 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

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's National Research Institute: lead

Study Sites (1)

Children's National Health System

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

Location

Related Publications (51)

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Biospecimen

Retention: SAMPLES WITHOUT DNA

An additional 5cc of blood work will be drawn at the time that the intravascular catheter is inserted for contrast agent administration for the cardiac MRI scans. This blood will be used to identify serum biomarkers in myocardial fibrosis, and to validate the potential use for diagnostic purposes. No genetic testing will be done on these samples. The blood samples will be identified with the study ID number only, and will be stored in a secure freezer.

MeSH Terms

Conditions

Muscular Dystrophies; Genetic Diseases, Inborn; Musculoskeletal Diseases

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Christopher Spurney, MD

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: August 29, 2016
• First Posted: October 3, 2016
• Study Start: January 1, 2014
• Primary Completion: December 9, 2024
• Study Completion: December 9, 2024
• Last Updated: December 11, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)