NCT01873976

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.

Trial Health

47
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
288

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jun 2013

Longer than P75 for all trials

Geographic Reach
2 countries

13 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2013

Completed
5 days until next milestone

First Submitted

Initial submission to the registry

June 6, 2013

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 10, 2013

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2017

Completed
5.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2022

Completed
Last Updated

October 19, 2020

Status Verified

October 1, 2020

Enrollment Period

3.6 years

First QC Date

June 6, 2013

Last Update Submit

October 14, 2020

Conditions

Dilated CardiomyopathyHypertrophic Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

  • Time to Death

    2 years

Secondary Outcomes (2)

  • Time to heart transplant

    2 years

  • Worsening heart failure

    2 years

Study Arms (3)

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.

Eligibility Criteria

AgeUp to 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Pediatric cases of dilated and hypertrophic cardiomyopathy will be recruited at 11 pediatric cardiology centers in the US and Canada.

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • Malignancy
  • Systemic Hypertension
  • Pulmonary parenchymal or vascular disease (e.g., cystic fibrosis, cor pulmonale, or pulmonary hypertension)
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Ann and Robert H. Lurie Children's Hospital

Chicago, Illinois, 60611, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Children's Hospital of New York, Columbia Presbyterian Medical Center

New York, New York, 10032, United States

Location

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh of UMPC

Pittsburgh, Pennsylvania, 15224, United States

Location

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103, United States

Location

Monroe Carell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

Location

Primary Children's Medical Center

Salt Lake City, Utah, 84113, United States

Location

Stollery Children's Hospital, University of Alberta

Edmonton, Alberta, T6G 2B7, Canada

Location

Related Publications (3)

  • Schmitt W, Diedrich C, Hamza TH, Meyer M, Eissing T, Breitenstein S, Rossano JW, Lipshultz SE. NT-proBNP for Predicting All-Cause Death and Heart Transplant in Children and Adults with Heart Failure. Pediatr Cardiol. 2025 Mar;46(3):694-703. doi: 10.1007/s00246-024-03489-7. Epub 2024 May 9.

    PMID: 38722325DERIVED

  • Kirmani S, Woodard PK, Shi L, Hamza TH, Canter CE, Colan SD, Pahl E, Towbin JA, Webber SA, Rossano JW, Everitt MD, Molina KM, Kantor PF, Jefferies JL, Feingold B, Addonizio LJ, Ware SM, Chung WK, Ballweg JA, Lee TM, Bansal N, Razoky H, Czachor J, Lunze FI, Marcus E, Commean P, Wilkinson JD, Lipshultz SE. Cardiac imaging and biomarkers for assessing myocardial fibrosis in children with hypertrophic cardiomyopathy. Am Heart J. 2023 Oct;264:153-162. doi: 10.1016/j.ahj.2023.06.005. Epub 2023 Jun 12.

    PMID: 37315879DERIVED

  • Everitt MD, Wilkinson JD, Shi L, Towbin JA, Colan SD, Kantor PF, Canter CE, Webber SA, Hsu DT, Pahl E, Addonizio LJ, Dodd DA, Jefferies JL, Rossano JW, Feingold B, Ware SM, Lee TM, Godown J, Simpson KE, Sleeper LA, Czachor JD, Razoky H, Hill A, Westphal J, Molina KM, Lipshultz SE; Pediatric Cardiomyopathy Registry Investigators. Cardiac Biomarkers in Pediatric Cardiomyopathy: Study Design and Recruitment Results from the Pediatric Cardiomyopathy Registry. Prog Pediatr Cardiol. 2019 Jun;53:1-10. doi: 10.1016/j.ppedcard.2019.02.004. Epub 2019 Mar 7.

    PMID: 31745384DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma, Serum

MeSH Terms

Conditions

Cardiomyopathy, DilatedCardiomyopathy, Hypertrophic

Condition Hierarchy (Ancestors)

CardiomegalyHeart DiseasesCardiovascular DiseasesCardiomyopathiesLaminopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAortic Stenosis, SubvalvularAortic Valve StenosisAortic Valve DiseaseHeart Valve Diseases

Study Officials

  • Steven E Lipshultz, MD

    Wayne State University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
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

Study Record Dates

First Submitted

June 6, 2013

First Posted

June 10, 2013

Study Start

June 1, 2013

Primary Completion

January 1, 2017

Study Completion

June 1, 2022

Last Updated

October 19, 2020

Record last verified: 2020-10

Locations

US(12)CA(1)