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Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02921321
Recruitment Status : Recruiting
First Posted : October 3, 2016
Last Update Posted : January 18, 2018
Sponsor:
Information provided by (Responsible Party):

August 29, 2016
October 3, 2016
January 18, 2018
January 2014
August 2023   (Final data collection date for primary outcome measure)
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. ]
Same as current
Complete list of historical versions of study NCT02921321 on ClinicalTrials.gov Archive Site
  • 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. ]
  • 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 ]
  • 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. ]
Same as current
Not Provided
Not Provided
 
Pilot Study of Cardiac MR in Patients With Muscular Dystrophy
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.
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.
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples Without DNA
Description:
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.
Non-Probability Sample
Male or females at least 6 years of age with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy.
  • Muscular Dystrophies
  • Cardiac Fibrosis
  • Genetic Diseases, Inborn
  • Musculoskeletal Diseases
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
100
December 2023
August 2023   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
6 Years and older   (Child, Adult, Senior)
No
Contact: Christopher Spurney, MD 202-476-2020 cspurney@childrensnational.org
Contact: Karin Hamann, RN 202-476-2317 khamann@childrensnational.org
United States
 
 
NCT02921321
4193
Internal Bridge Funding ( Other Identifier: Children's Research Institute,Children's National )
No
Not Provided
Plan to Share IPD: No
Christopher Spurney, Children's Research Institute
Children's Research Institute
Not Provided
Principal Investigator: Christopher Spurney, MD Children's Research Institute
Children's Research Institute
January 2018