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Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
New England Research Institutes
Washington University School of Medicine
Boston Children’s Hospital
Columbia University
Children's Hospital of Philadelphia
Ann & Robert H Lurie Children's Hospital of Chicago
Primary Children's Hospital
Monroe Carell Jr. Children's Hospital at Vanderbilt
Stollery Children's Hospital
Children's Hospital Medical Center, Cincinnati
Montefiore Medical Center
Children's Hospital Colorado
Le Bonheur Children's Hospital
Children's Hospital of Pittsburgh
Information provided by (Responsible Party):
Steve Lipshultz, Wayne State University
ClinicalTrials.gov Identifier:
NCT01873976
First received: June 6, 2013
Last updated: May 19, 2016
Last verified: May 2016
  Purpose
Cardiomyopathy is a disease of the heart muscle. It is rare, but it can be serious. Cardiomyopathy in children can result in death, disability, heart transplantation or serious heart rhythm disorders. Natural substances in the blood called cardiac biomarkers can be measured in the laboratory and could be a less invasive way (compared to echocardiograms or MRIs) to detect heart dysfunction in children with cardiomyopathy. Little is known about how useful and valid cardiac biomarkers are in the diagnosis and determination of the symptoms in children with cardiomyopathy. The long-term goal of this project is to study how helpful measuring cardiac biomarkers in children with cardiomyopathy is to their doctors in managing the care of these patients as well as improving their overall health. Measures of these cardiac biomarkers could help doctors in determining how best to care for a child with cardiomyopathy, including when to consider heart transplantation as a treatment option.

Condition
Dilated Cardiomyopathy
Hypertrophic Cardiomyopathy

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Cardiac Biomarkers in Pediatric Cardiomyopathy

Resource links provided by NLM:


Further study details as provided by Wayne State University:

Primary Outcome Measures:
  • Time to Death [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Time to heart transplant [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Worsening heart failure [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples Without DNA
Plasma, Serum

Enrollment: 288
Study Start Date: June 2013
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts
Incident DCM
A case of dilated cardiomyopathy that presents to the study site for the first time.
Incident/Recent HCM
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.

Detailed Description:

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:

  1. 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)
  2. 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.
  3. 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.

  Eligibility

Ages Eligible for Study:   up to 20 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Pediatric cases of dilated and hypertrophic cardiomyopathy will be recruited at 11 pediatric cardiology centers in the US and Canada.
Criteria

Inclusion Criteria:

  • Patient is alive and has not received a transplant prior to enrollment in the study.
  • Under age 21 years at age of enrollment
  • For Group 1 (incident DCM), a case of DCM presenting to a study site within 2 years of the original cardiomyopathy diagnosis
  • Group 2 (incident/recent HCM), a new or existing diagnosis of idiopathic or familial HCM with a cMRI within 12 months of diagnosis
  • Group 3 (prevalent HCM or DCM), any child with a diagnosis of DCM or idiopathic, familial, or HCM due to a known disease-causing mutation who has survived transplant-free at least 12 months from the date of original cardiomyopathy diagnosis
  • For all 3 groups, diagnosis of cardiomyopathy must be confirmed by Echocardiographic or cMRI criteria

Exclusion Criteria:

A patient is not eligible for enrollment if one or more of the following conditions are met at the time of presentation with cardiomyopathy:

  • Any cardiomyopathy diagnosis other than DCM or idiopathic HCM, familial HCM or HCM due to a known disease-causing gene
  • Endocrine disease known to cause heart muscle disease (including infants of diabetic mothers)
  • History of rheumatic fever
  • Toxic exposures known to cause heart muscle disease (anthracyclines, mediastinal radiation, iron overload or heavy metal exposure)
  • HIV infection or born to an HIV positive mother
  • Kawasaki disease
  • Congenital heart defects unassociated with malformation syndromes (e.g., valvular heart disease or congenital coronary artery malformations)
  • Immunologic disease
  • Invasive cardiothoracic procedures or major surgery during the preceding month, except those specifically related to cardiomyopathy including left ventricular assist device (LVAD), extracorporeal membrane oxygenator (ECMO), and automatic implantable cardioverter defibrillator (AICD) placement
  • Uremia, active or chronic
  • Abnormal ventricular size or function that can be attributed to intense physical training or chronic anemia
  • Chronic arrhythmia, unless there are studies documenting inclusion criteria prior to the onset of arrhythmia (except a patient with chronic arrhythmia, subsequently ablated, whose cardiomyopathy persists after two months is not to be excluded)
  • Malignancy
  • Systemic Hypertension
  • Pulmonary parenchymal or vascular disease (e.g., cystic fibrosis, cor pulmonale, or pulmonary hypertension)
  • Ischemic coronary vascular disease
  • Association with drugs (e.g., growth hormone, corticosteroids, cocaine) or other diseases known to cause hypertrophy
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01873976

Locations
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
St. 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
Canada, Alberta
Stollery Children's Hospital, University of Alberta
Edmonton, Alberta, Canada, T6G 2B7
Sponsors and Collaborators
Wayne State University
New England Research Institutes
Washington University School of Medicine
Boston Children’s Hospital
Columbia University
Children's Hospital of Philadelphia
Ann & Robert H Lurie Children's Hospital of Chicago
Primary Children's Hospital
Monroe Carell Jr. Children's Hospital at Vanderbilt
Stollery Children's Hospital
Children's Hospital Medical Center, Cincinnati
Montefiore Medical Center
Children's Hospital Colorado
Le Bonheur Children's Hospital
Children's Hospital of Pittsburgh
Investigators
Principal Investigator: Steven E Lipshultz, MD Wayne State University
  More Information

Responsible Party: Steve Lipshultz, Schotanus Family Endowed Chair of Pediatrics, Professor and Chair, Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine; President, University Pediatricians and Pediatrician-in-Chief, Children's Hospital of Michigan, Wayne State University
ClinicalTrials.gov Identifier: NCT01873976     History of Changes
Other Study ID Numbers: 1R01HL109090-01A1 
Study First Received: June 6, 2013
Last Updated: May 19, 2016
Health Authority: United States: Data and Safety Monitoring Board
United States: Institutional Review Board

Additional relevant MeSH terms:
Cardiomyopathies
Hypertrophy
Cardiomyopathy, Dilated
Cardiomyopathy, Hypertrophic
Heart Diseases
Cardiovascular Diseases
Pathological Conditions, Anatomical
Cardiomegaly
Aortic Stenosis, Subvalvular
Aortic Valve Stenosis
Heart Valve Diseases

ClinicalTrials.gov processed this record on September 26, 2016