Heart Imaging in Children With Muscular Dystrophy

NCT01633242 | Terminated

• 2 other identifiers: 12009012-H-0090
• Study Type: observational
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

Background: \- Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function. However, better types of heart imaging studies are needed to improve treatment of heart problems related to muscular dystrophy. Better heart imaging methods are especially needed for children with muscular dystrophy. Researchers want to test different heart imaging methods in children with muscular dystrophy. They will look at cardiac magnetic resonance imaging (MRI) and standard heart function tests. Objectives: \- To develop and test new methods for imaging the heart in children with muscular dystrophy. Eligibility: \- Children and adolescents between 8 and 17 years of age who have muscular dystrophy. Design:

• Participants will be screened with a physical exam and medical history.
• Participants will provide a blood sample at the start of the study. They will also have heart function tests before having the imaging study.
• Participants will have a cardiac MRI scan that will last up to 60 minutes. Some tests will require a MRI contrast agent (a drug that helps the image appear more clearly on the scan).

Conditions

Muscular Dystrophy

Keywords

MRI; Muscular Dystrophy; Cardiac; Myocardium; Intramyocardial fat

Outcome Measures

Primary Outcomes (1)

• Evaluate CMR measures in muscular dystrophy patients

Secondary Outcomes (3)

• Early Detection

• Measurement of Disease Progression

• Ultimate identification of the response to developing therapies

Eligibility Criteria

• Age: 8 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or females with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy.
• to 100 years of age.

You may not qualify if:

• Day-time users of continuous positive airway pressure (CPAP)
• Sip ventilator users
• Invasive ventilator dependent
• Pregnant minors (when uncertain, participants will undergo urine testing) or lactating minors
• Decompensated congestive heart failure (unable to lie flat during CMR)
• Impaired renal excretory function (calculated Glomerular Filtration Rate less than 30mL/min)
• Contra-indications to Magnetic Resonance Imaging:
• Cardiac pacemaker or implantable defibrillator
• Cerebral aneurysm clip
• Neural stimulator
• Metallic ocular foreign body
• Harrington-rod
• Any implanted device (i.e. insulin pump, drug infusion device)
• Claustrophobia
• Metal shrapnel or bullet

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 Dec 24;51(6):919-28. doi: 10.1016/0092-8674(87)90579-4.

  PMID: 3319190 BACKGROUND

• Nigro G, Comi LI, Politano L, Bain RJ. The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy. Int J Cardiol. 1990 Mar;26(3):271-7. doi: 10.1016/0167-5273(90)90082-g.

  PMID: 2312196 BACKGROUND

• Corrado G, Lissoni A, Beretta S, Terenghi L, Tadeo G, Foglia-Manzillo G, Tagliagambe LM, Spata M, Santarone M. Prognostic value of electrocardiograms, ventricular late potentials, ventricular arrhythmias, and left ventricular systolic dysfunction in patients with Duchenne muscular dystrophy. Am J Cardiol. 2002 Apr 1;89(7):838-41. doi: 10.1016/s0002-9149(02)02195-1.

  PMID: 11909570 BACKGROUND

MeSH Terms

Conditions

Muscular Dystrophies

Condition Hierarchy (Ancestors)

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

Study Officials

• Andrew E Arai, M.D.

  National Heart, Lung, and Blood Institute (NHLBI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2012
• First Posted: July 4, 2012
• Study Start: February 24, 2012
• Study Completion: October 11, 2016
• Last Updated: September 13, 2019

Record last verified: 2016-10-11

Locations

US (1)