Dilated CardiomyopathyNCT01873976
Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers)
An observational study of Dilated Cardiomyopathy and Hypertrophic Cardiomyopathy, run by Wayne State University at 13 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 20 years
- Conditions the study is for
- Dilated Cardiomyopathy, Hypertrophic Cardiomyopathy
- How many people
- 288 participants
- Where
- 13 sitesin 2 countries
- Study dates
- Jun 2013to Jun 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric cases of dilated and hypertrophic cardiomyopathy will be recruited at 11 pediatric cardiology centers in the US and Canada.
Who may be able to join
- 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
Who cannot join
- 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
Show all 18Show fewer
- 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
Trial locations and contacts
13 sites in 2 countries.
13 sites in 2 countries
United States12 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Children's Hospital Boston
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
Children's Hospital of New York, Columbia Presbyterian Medical Center
New York, New York, 10032
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UMPC
Pittsburgh, Pennsylvania, 15224
Primary Children's Medical Center
Salt Lake City, Utah, 84113
Washington University School of Medicine
St Louis, Missouri, 63110
Children's Hospital at Montefiore
The Bronx, New York, 10467
Canada1 site
Stollery Children's Hospital, University of Alberta
Edmonton, Alberta, T6G 2B7
About this study
Official title: Cardiac Biomarkers in Pediatric Cardiomyopathy
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.
Read the 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:
- 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.
- Medical subject headings
- Cardiomyopathy, Dilated, Cardiomyopathy, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Death
Measured: 2 years
Secondary outcomes (2)
Time to heart transplant
Measured: 2 years
Worsening heart failure
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2013 (estimated)
- Study startJun 2013
- Primary completionJan 2017
- Study completionJun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Dilated CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 288 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wayne State University
- Sponsor type
- University, hospital or other
- Collaborators
- Carelon Research, 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, University of Pittsburgh
- Responsible party
- Principal investigator, Steve Lipshultz, Wayne State University
- Investigators
- Steven E Lipshultz, MD, Principal investigator, Wayne State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01873976
- Sponsor's protocol ID
- 1R01HL109090-01A1
- Status last verified
- Oct 2020
- First submitted
- 6 Jun 2013
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 38722325Kirmani 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.
PubMed 37315879Everitt 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.
PubMed 31745384
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cardiac Biomarkers in Pediatric Cardiomyopathy
- ClinicalTrials.gov ID
- NCT01873976
- Acronym / sponsor ID
- 1R01HL109090-01A1
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 288 participants (actual)
- Lead sponsor
- Wayne State UniversityUniversity, hospital or other
- Collaborators
- Carelon Research, 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, University of Pittsburgh
- Study start
- Jun 2013 (actual)
- Primary completion
- Jan 2017 (actual)
- Study completion
- Jun 2019 (actual)
- First posted
- 10 Jun 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- Up to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric cases of dilated and hypertrophic cardiomyopathy will be recruited at 11 pediatric cardiology centers in the US and Canada.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (18 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Time to Death
Time frame: 2 years
2 secondary outcomes
Time to heart transplant
Time frame: 2 years
Worsening heart failure
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2026.
- Derived from this study: PubMed 38722325Schmitt 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.
- Derived from this study: PubMed 37315879Kirmani 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.
- Derived from this study: PubMed 31745384Everitt 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven E Lipshultz, MD principal investigator
Wayne State University
13 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Dilated Cardiomyopathy and Hypertrophic Cardiomyopathy, run by Wayne State University at 13 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to Death (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01873976, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2026.