Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01873976|
Recruitment Status : Active, not recruiting
First Posted : June 10, 2013
Last Update Posted : October 19, 2020
|Condition or disease|
|Dilated Cardiomyopathy Hypertrophic Cardiomyopathy|
Pediatric cardiomyopathy is a heterogeneous disease with high morbidity and mortality in which children often present with fulminant disease leading to death or transplant. Highly sensitive and specific cardiac biomarkers or panels of biomarkers, representing different pathologic mechanisms or pathways, are the least invasive (a particularly important consideration in children) and most-cost-effective approach to the early detection of cardiac dysfunction. The long-term goal of this project is to identify such biomarkers in children with cardiomyopathy. These findings could represent a major advance in determining the most appropriate evidence-based clinical care for these children, including when to consider heart transplantation.
The specific aims of this study are:
- To determine the ability of established and novel cardiac biomarkers to predict short- and long-term outcomes in children with newly diagnosed (incident) dilated cardiomyopathy (DCM)
- To assess the clinical utility of cardiac biomarkers of collagen metabolism in determining the presence of myocardial fibrosis, as established by cardiac MRI (cMRI), and left ventricular (LV) diastolic dysfunction, as established by echocardiography in both newly diagnosed and existing cases of idiopathic and familial hypertrophic cardiomyopathy (HCM) in children.
- To determine whether longitudinal changes in cardiac biomarkers are associated with worsening heart failure (HF) class in clinically stable children with dilated or hypertrophic cardiomyopathy. This study will determine the importance of serological biomarkers, in conjunction with cardiac imaging, in the early identification of heart disease before cardiac remodeling and functional impairment have become irreversible in a pediatric population.
This is a 5-year prospective study of up to 480 children with either primary dilated or hypertrophic cardiomyopathy. The study will have three components: 1) clinical data collection by chart review, 2) biospecimen collection and testing, and 3) centralized review and measurement of echocardiograms and cMRIs.
|Study Type :||Observational|
|Actual Enrollment :||288 participants|
|Official Title:||Cardiac Biomarkers in Pediatric Cardiomyopathy|
|Actual Study Start Date :||June 2013|
|Actual Primary Completion Date :||January 2017|
|Estimated Study Completion Date :||June 2022|
A case of dilated cardiomyopathy that presents to the study site for the first time.
A new or existing diagnosis of idiopathic or familial hypertrophic cardiomyopathy, diagnosed within the past 2 months and with a cMRI within 2 months of diagnosis.
Prevalent HCM or DCM
Any child with a diagnosis of dilated cardiomyopathy or idiopathic or familial hypertrophic cardiomyopathy who has survived transplant-free at least 24 months from the date of cardiomyopathy diagnosis.
- Time to Death [ Time Frame: 2 years ]
- Time to heart transplant [ Time Frame: 2 years ]
- Worsening heart failure [ Time Frame: 2 years ]
Biospecimen Retention: Samples Without DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01873976
|United States, Colorado|
|Children's Hospital Colorado|
|Aurora, Colorado, United States, 80045|
|United States, Illinois|
|Ann and Robert H. Lurie Children's Hospital|
|Chicago, Illinois, United States, 60611|
|United States, Massachusetts|
|Children's Hospital Boston|
|Boston, Massachusetts, United States, 02115|
|United States, Missouri|
|Washington University School of Medicine|
|Saint Louis, Missouri, United States, 63110|
|United States, New York|
|Children's Hospital at Montefiore|
|Bronx, New York, United States, 10467|
|Children's Hospital of New York, Columbia Presbyterian Medical Center|
|New York, New York, United States, 10032|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Children's Hospital of Pittsburgh of UMPC|
|Pittsburgh, Pennsylvania, United States, 15224|
|United States, Tennessee|
|Le Bonheur Children's Hospital|
|Memphis, Tennessee, United States, 38103|
|Monroe Carell Jr. Children's Hospital at Vanderbilt|
|Nashville, Tennessee, United States, 37232|
|United States, Utah|
|Primary Children's Medical Center|
|Salt Lake City, Utah, United States, 84113|
|Stollery Children's Hospital, University of Alberta|
|Edmonton, Alberta, Canada, T6G 2B7|
|Principal Investigator:||Steven E Lipshultz, MD||Wayne State University|