Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers)

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by University of Miami
Sponsor:
Collaborators:
New England Research Institutes
Washington University School of Medicine
Children's Hospital Boston
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
Information provided by (Responsible Party):
Steven E. Lipshultz, MD, University of Miami
ClinicalTrials.gov Identifier:
NCT01873976
First received: June 6, 2013
Last updated: May 5, 2014
Last verified: May 2014
  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 University of Miami:

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


Estimated Enrollment: 480
Study Start Date: June 2013
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: December 2015 (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
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 2 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 24 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

Contacts
Contact: Steven E Lipshultz, MD 305-243-3993 SLipshultz@med.miami.edu
Contact: James D Wilkinson, MD, MPH jwilkins@med.miami.edu

Locations
United States, Florida
University of Miami/Jackson Memorial Hospital Not yet recruiting
Miami, Florida, United States, 33136
Contact: Paolo Rusconi, MD       prusconi@med.miami.edu   
Principal Investigator: Paolo Rusconi, MD         
United States, Illinois
Ann and Robert H. Lurie Children's Hospital Recruiting
Chicago, Illinois, United States, 60611
Contact: Elfriede Pahl, MD       EPahl@luriechildrens.org   
Principal Investigator: Elfriede Pahl, MD         
United States, Massachusetts
Children's Hospital Boston Recruiting
Boston, Massachusetts, United States, 02115
Contact: Steven Colan, MD       Steven.Colan@cardio.chboston.org   
Principal Investigator: Steven Colan, MD         
United States, Missouri
Washington University School of Medicine Recruiting
St. Louis, Missouri, United States, 63110
Contact: Charles Canter, MD       canter@kids.wustl.edu   
Principal Investigator: Charles Canter, MD         
United States, New York
Children's Hospital at Montefiore Not yet recruiting
Bronx, New York, United States, 10467
Contact: Daphne Hsu, MD       dhsu@montefiore.org   
Principal Investigator: Daphne Hsu, MD         
Children's Hospital of New York, Columbia Presbyterian Medical Center Recruiting
New York, New York, United States, 10032
Contact: Linda Addonizio, MD       lja1@mail.cumc.columbia.edu   
Principal Investigator: Linda Addonizio, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Jeffrey Towbin, MD       jeffrey.towbin@cchmc.org   
Principal Investigator: Jeffrey Towbin, MD         
Sub-Investigator: John L Jeffries, MD, MPH         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Joseph Rossano, MD, MS       RossanoJ@email.chop.edu   
Principal Investigator: Joseph Rossano, MD MS         
United States, Tennessee
Monroe Carell Jr. Children's Hospital at Vanderbilt Recruiting
Nashville, Tennessee, United States, 37232
Contact: Steven Webber, MBChB, MRCP       steve.a.webber@vanderbilt.edu   
Principal Investigator: Steven Webber, MBChB, MRCP         
Sub-Investigator: Debra Dodd, MD         
United States, Utah
Primary Children's Medical Center Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Melanie Everitt, MD       Melanie.Everitt@imail.org   
Principal Investigator: Melanie Everitt, MD         
Canada, Alberta
Stollery Children's Hospital, University of Alberta Recruiting
Edmonton, Alberta, Canada, T6G 2B7
Contact: Paul Kantor, MBBCh, FRCPC       paul.kantor@albertahealthservices.ca   
Principal Investigator: Paul Kantor, MBBCh, FRCPC         
Sponsors and Collaborators
University of Miami
New England Research Institutes
Washington University School of Medicine
Children's Hospital Boston
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
Investigators
Principal Investigator: Steven E Lipshultz, MD University of Miami
  More Information

No publications provided

Responsible Party: Steven E. Lipshultz, MD, George Batchelor Professor of Pediatrics, Professor of Epidemiology and Public Health, Professor of Medicine (Oncology); Director, Batchelor Children's Research Institute, University of Miami
ClinicalTrials.gov Identifier: NCT01873976     History of Changes
Other Study ID Numbers: 1R01HL109090-01A1
Study First Received: June 6, 2013
Last Updated: May 5, 2014
Health Authority: United States: Data and Safety Monitoring Board
United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on July 29, 2014