VVV: Variability of Ventricular Mass, Volume, & Ejection Fraction in Pediatric Cardiomyopathy Patients-Pediatric Heart Network
Study Details
Study Description
Brief Summary
This observational study will provide data (variations in ventricular size and function) that are essential to designing and conducting clinical trials. In addition, the study will evaluate intra- and inter-study variability seen in echocardiography.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Detailed Description
BACKGROUND:
Cardiomyopathy is an important cause of chronic disability and death in pediatric patients and currently accounts for approximately 50% of cardiac transplants performed during childhood. Left ventricular (LV) size and function are important independent predictors of outcome, and echocardiography is the primary way to assess ventricular function in children. Although there is extensive experience with this technology, data are limited on how ventricular function changes over time in children, which is a major impediment to conducting controlled trials of therapy in children.
This study has been approved by the Institutional Review Boards/Research Ethics Boards of all participating clinical centers:
Hospital for Sick Children, Toronto, Canada
Children's Hospital Boston, Boston, MA
Columbia College of Physicians and Surgeons, New York, NY
Children's Hospital of Philadelphia, Philadelphia, PA
Duke University Medical Center, Durham, NC
Brody School of Medicine at East Carolina University, Greenville, NC
Wake Forest Baptist Medical Center, Winston Salem, NC
Medical University of South Carolina, Charleston, SC
Primary Children's Medical Center, Salt Lake City, UT
Washington University, St. Louis, MO
DESIGN NARRATIVE:
This observational study will prospectively evaluate pediatric patients with dilated cardiomyopathy who are undergoing clinically indicated echocardiographic evaluation of LV function. Patients who undergo at least two echocardiograms 3 to 18 months apart will have their studies performed by the same ultrasonographer. All studies will be sent to the core laboratory for evaluation of variability in LV mass, volume, and ejection fraction.
Study Design
Outcome Measures
Primary Outcome Measures
- Inter-study variability of echo measurements; variance at a single point in time and variance of change in measurements over time [Measured for up to 18 months]
Secondary Outcome Measures
- Relative magnitude of the various sources of variability in echocardiographic outcomes in order to optimize operational procedures that can minimize variance [Measured for up to 18 months]
- Interstudy variability of echocardiographically-derived indices of LV systolic and diastolic function derived from m-mode, spectral Doppler, and tissue Doppler techniques used in pediatric patients with dilated cardiomyopathy [Measured for up to 18 months]
- Relationship of clinical status, including treatment, to the interstudy variability and repeatability of echocardiographic measurements. [Measured for up to 18 months]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Less than 22 years of age
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Any race
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Diagnosis of dilated cardiomyopathy
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LV and diastolic diameter greater than 5.5 cm (or z-score for BSA greater than 2) on the primary image acquisition from the first study echocardiogram
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LV ejection fraction less than 50% (or z-score for age less than -2) or shortening fraction less than 28% (or z-score for age less than -2) as measured on the primary image acquisition from the first study echocardiogram
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Disease onset greater than 2 months prior to screening
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Anticipated to undergo repeat evaluation at the same institution at least 3 months but not more than 13 months later
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Informed consent of parent(s) or legal guardian and assent of subject if required
Exclusion Criteria:
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Hypertrophic cardiomyopathy
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Restrictive cardiomyopathy
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Myocardial noncompaction (LV hypertrabeculation); patient is eligible for the study as long as the echocardiogram performed at the time of screening has no evidence of myocardial noncompaction
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Ventricular paced rhythm
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Atrial or ventricular ectopy at ratio greater than 1:4
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Suspected acute myocarditis
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Tachycardia-induced cardiomyopathy
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Congenital heart disease (repaired or unrepaired)
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Currently on intravenous inotropic support
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Current left ventricular assist device (LVAD) or extracorporeal membrane oxygenation (ECMO)
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Heart transplant waiting list status of 1A or 1B
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Co-morbid condition that precludes the ability to successfully obtain an echocardiogram according to the specifications of the study protocol
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Hospital Boston | Boston | Massachusetts | United States | 02115 |
| 2 | Washington University | St. Louis | Missouri | United States | 63110 |
| 3 | Columbia College of Physicians and Surgeons | New York | New York | United States | 10032 |
| 4 | Duke University Medical Center | Durham | North Carolina | United States | 27710 |
| 5 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
| 6 | Medical University of South Carolina | Charleston | South Carolina | United States | 29425 |
| 7 | Primary Children's Hospital | Salt Lake City | Utah | United States | 84132 |
| 8 | Hospital for Sick Children | Toronto | Ontario | Canada | M5G 1X8 |
Sponsors and Collaborators
- HealthCore-NERI
- National Heart, Lung, and Blood Institute (NHLBI)
- Pediatric Heart Network
Investigators
- Principal Investigator: Lynn Sleeper, ScD, HealthCore-NERI
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 236
- U01HL068292
- U01HL068290
- U01HL068288
- U01HL068285
- U01HL068281
- U01HL068279
- U01HL068270
- U42 HL068269