Heart Imaging in Children With Muscular Dystrophy
| Status: | Terminated |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 8 - 100 |
| Updated: | 4/21/2017 |
| Start Date: | February 24, 2012 |
| End Date: | October 11, 2016 |
A Pilot Study of Cardiac Magnetic Resonance in Patients With Muscular Dystrophy
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).
- 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).
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.
Using the framework of the Cooperative International Neuromuscular Research Group (CINRG)
and the Clinical and Translational Science Award (CTSA) consortium, this pilot study will
assess cardiac outcome measures in children obtained by gadolinium enhanced cardiac Magnetic
Resonance Imaging (MRI) and echocardiographic methods that can be reliably implemented
across a consortium of clinical sites devoted to the study of pharmaceutical treatments for
muscular dystrophy. These cardiac MRI scans will be shared with the PITT1109 research
protocol in all subjects that are enrolled in both studies.
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.
Using the framework of the Cooperative International Neuromuscular Research Group (CINRG)
and the Clinical and Translational Science Award (CTSA) consortium, this pilot study will
assess cardiac outcome measures in children obtained by gadolinium enhanced cardiac Magnetic
Resonance Imaging (MRI) and echocardiographic methods that can be reliably implemented
across a consortium of clinical sites devoted to the study of pharmaceutical treatments for
muscular dystrophy. These cardiac MRI scans will be shared with the PITT1109 research
protocol in all subjects that are enrolled in both studies.
- INCLUSION CRITERIA:
- Male or females with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular
Dystrophy.
- 8 to 100 years of age.
EXCLUSION CRITERIA:
- 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
- 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
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
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