Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers)

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Brief Title

Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers)

Official Title

Cardiac Biomarkers in Pediatric Cardiomyopathy

Brief Summary

      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.
    

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.
    


Study Type

Observational


Primary Outcome

Time to Death

Secondary Outcome

 Time to heart transplant

Condition

Dilated Cardiomyopathy


Study Arms / Comparison Groups

 Incident DCM
Description:  A case of dilated cardiomyopathy that presents to the study site for the first time.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

288

Start Date

June 2013

Completion Date

June 2020

Primary Completion Date

January 2017

Eligibility 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
      

Gender

All

Ages

N/A - 20 Years

Accepts Healthy Volunteers

No

Contacts

Steven E Lipshultz, MD, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT01873976

Organization ID

1R01HL109090-01A1


Responsible Party

Principal Investigator

Study Sponsor

Wayne State University

Collaborators

 HealthCore-NERI

Study Sponsor

Steven E Lipshultz, MD, Principal Investigator, Wayne State University


Verification Date

October 2019