Cardiomyopathy, DilatedVVVNCT00123071
Variability of Ventricular Mass, Volume, & Ejection Fraction in Pediatric Cardiomyopathy Patients-Pediatric Heart Network
An observational study of Cardiomyopathy, Dilated, run by Carelon Research at 8 sites in 2 countries.
Trial summary
This observational study will provide data (variations in ventricular size and function) that are essential to designing and conducting clinical trials. In addition, the study will evaluate intra- and inter-study variability seen in echocardiography. Read more about this study
- Who is it for?
- Ages Up to 22 years
- Conditions the study is for
- Cardiomyopathy, Dilated
- How many people
- 131 participants
- Where
- 8 sitesin 2 countries
- Study dates
- May 2005to Jun 2009
- 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 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients up to 22 years of age with dilated cardiomyopathy followed at participating clinical centers or newly diagnosed during the recruitment period
Who may be able to join
- Less than 22 years of age
- Any race
- Diagnosis of dilated cardiomyopathy
- LV and diastolic diameter greater than 5.5 cm (or z-score for BSA greater than 2) on the primary image acquisition from the first study echocardiogram
- LV ejection fraction less than 50% (or z-score for age less than -2) or shortening fraction less than 28% (or z-score for age less than -2) as measured on the primary image acquisition from the first study echocardiogram
- Disease onset greater than 2 months prior to screening
- Anticipated to undergo repeat evaluation at the same institution at least 3 months but not more than 13 months later
- Informed consent of parent(s) or legal guardian and assent of subject if required
Who cannot join
- Hypertrophic cardiomyopathy
- Restrictive cardiomyopathy
- Myocardial noncompaction (LV hypertrabeculation); patient is eligible for the study as long as the echocardiogram performed at the time of screening has no evidence of myocardial noncompaction
- Ventricular paced rhythm
- Atrial or ventricular ectopy at ratio greater than 1:4
- Suspected acute myocarditis
Show all 12Show fewer
- Tachycardia-induced cardiomyopathy
- Congenital heart disease (repaired or unrepaired)
- Currently on intravenous inotropic support
- Current left ventricular assist device (LVAD) or extracorporeal membrane oxygenation (ECMO)
- Heart transplant waiting list status of 1A or 1B
- Co-morbid condition that precludes the ability to successfully obtain an echocardiogram according to the specifications of the study protocol
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States7 sites
Children's Hospital Boston
Boston, Massachusetts, 02115
Medical University of South Carolina
Charleston, South Carolina, 29425
Duke University Medical Center
Durham, North Carolina, 27710
Columbia College of Physicians and Surgeons
New York, New York, 10032
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Primary Children's Hospital
Salt Lake City, Utah, 84132
Washington University
St Louis, Missouri, 63110
About this study
Official title: Variability of Ventricular Mass, Volume, and Ejection Fraction in Pediatric Cardiomyopathy Patients (A Study Conducted by the Pediatric Heart Network)
This observational study will provide data (variations in ventricular size and function) that are essential to designing and conducting clinical trials. In addition, the study will evaluate intra- and inter-study variability seen in echocardiography.
Read the detailed description
BACKGROUND:
Cardiomyopathy is an important cause of chronic disability and death in pediatric patients and currently accounts for approximately 50% of cardiac transplants performed during childhood. Left ventricular (LV) size and function are important independent predictors of outcome, and echocardiography is the primary way to assess ventricular function in children. Although there is extensive experience with this technology, data are limited on how ventricular function changes over time in children, which is a major impediment to conducting controlled trials of therapy in children.
This study has been approved by the Institutional Review Boards/Research Ethics Boards of all participating clinical centers:
Hospital for Sick Children, Toronto, Canada
Children's Hospital Boston, Boston, MA
Columbia College of Physicians and Surgeons, New York, NY
Children's Hospital of Philadelphia, Philadelphia, PA
Duke University Medical Center, Durham, NC
Brody School of Medicine at East Carolina University, Greenville, NC
Wake Forest Baptist Medical Center, Winston Salem, NC
Medical University of South Carolina, Charleston, SC
Primary Children's Medical Center, Salt Lake City, UT
Washington University, St. Louis, MO
DESIGN NARRATIVE:
This observational study will prospectively evaluate pediatric patients with dilated cardiomyopathy who are undergoing clinically indicated echocardiographic evaluation of LV function. Patients who undergo at least two echocardiograms 3 to 18 months apart will have their studies performed by the same ultrasonographer. All studies will be sent to the core laboratory for evaluation of variability in LV mass, volume, and ejection fraction.
- Conditions
- Cardiomyopathy, Dilated
- Medical subject headings
- Cardiomyopathy, Dilated
What is being measured
The primary outcome is the main question the study is designed to answer.
Inter-study variability of echo measurements; variance at a single point in time and variance of change in measurements over time
Measured: Measured for up to 18 months
Secondary outcomes (3)
Relative magnitude of the various sources of variability in echocardiographic outcomes in order to optimize operational procedures that can minimize variance
Measured: Measured for up to 18 months
Interstudy variability of echocardiographically-derived indices of LV systolic and diastolic function derived from m-mode, spectral Doppler, and tissue Doppler techniques used in pediatric patients with dilated cardiomyopathy
Measured: Measured for up to 18 months
Relationship of clinical status, including treatment, to the interstudy variability and repeatability of echocardiographic measurements.
Measured: Measured for up to 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2005 (estimated)
- Study startMay 2005
- Primary completionOct 2008
- Study completionJun 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Cardiomyopathy, DilatedStudies 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
- 131 participants (actual)
Sponsor and investigators
- Lead sponsor
- Carelon Research
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Pediatric Heart Network
- Responsible party
- Sponsor
- Investigators
- Lynn Sleeper, ScD, Principal investigator, Carelon Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00123071
- Sponsor's protocol ID
- 236
- Other IDs
- U01HL068292, U01HL068290, U01HL068288, U01HL068285, U01HL068281, U01HL068279, U01HL068270, U42 HL068269
- Acronym
- VVV
- Status last verified
- Jan 2013
- First submitted
- 20 Jul 2005
- Last updated
- 25 Jan 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Selamet Tierney ES, Hollenbeck-Pringle D, Lee CK, Altmann K, Dunbar-Masterson C, Golding F, Lu M, Miller SG, Molina K, Natarajan S, Taylor CL, Trachtenberg F, Colan SD; Pediatric Heart Network Investigators. Reproducibility of Left Ventricular Dimension Versus Area Versus Volume Measurements in Pediatric Patients With Dilated Cardiomyopathy. Circ Cardiovasc Imaging. 2017 Nov;10(11):e006007. doi: 10.1161/CIRCIMAGING.116.006007.
PubMed 29133477Lee CK, Margossian R, Sleeper LA, Canter CE, Chen S, Tani LY, Shirali G, Szwast A, Tierney ES, Campbell MJ, Golding F, Wang Y, Altmann K, Colan SD; Pediatric Heart Network Investigators. Variability of M-mode versus two-dimensional echocardiography measurements in children with dilated cardiomyopathy. Pediatr Cardiol. 2014 Apr;35(4):658-67. doi: 10.1007/s00246-013-0835-9. Epub 2013 Nov 22.
PubMed 24265000Molina KM, Shrader P, Colan SD, Mital S, Margossian R, Sleeper LA, Shirali G, Barker P, Canter CE, Altmann K, Radojewski E, Tierney ES, Rychik J, Tani LY; Pediatric Heart Network Investigators. Predictors of disease progression in pediatric dilated cardiomyopathy. Circ Heart Fail. 2013 Nov;6(6):1214-22. doi: 10.1161/CIRCHEARTFAILURE.113.000125. Epub 2013 Oct 16.
PubMed 24132734Colan SD, Shirali G, Margossian R, Gallagher D, Altmann K, Canter C, Chen S, Golding F, Radojewski E, Camitta M, Carboni M, Rychik J, Stylianou M, Tani LY, Selamet Tierney ES, Wang Y, Sleeper LA; Pediatric Heart Network Investigators. The ventricular volume variability study of the Pediatric Heart Network: study design and impact of beat averaging and variable type on the reproducibility of echocardiographic measurements in children with chronic dilated cardiomyopathy. J Am Soc Echocardiogr. 2012 Aug;25(8):842-854.e6. doi: 10.1016/j.echo.2012.05.004. Epub 2012 Jun 5.
PubMed 22677278
Study at a glance
From the registry record, last updated by the sponsor on 25 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Variability of Ventricular Mass, Volume, and Ejection Fraction in Pediatric Cardiomyopathy Patients (A Study Conducted by the Pediatric Heart Network)
- ClinicalTrials.gov ID
- NCT00123071
- Acronym / sponsor ID
- VVV / 236
- Conditions
- Cardiomyopathy, Dilated
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 131 participants (actual)
- Lead sponsor
- Carelon ResearchUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Pediatric Heart Network
- Study start
- May 2005
- Primary completion
- Oct 2008 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 22 Jul 2005 (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.
This observational study will provide data (variations in ventricular size and function) that are essential to designing and conducting clinical trials. In addition, the study will evaluate intra- and inter-study variability seen in echocardiography.
Detailed description
BACKGROUND:
Cardiomyopathy is an important cause of chronic disability and death in pediatric patients and currently accounts for approximately 50% of cardiac transplants performed during childhood. Left ventricular (LV) size and function are important independent predictors of outcome, and echocardiography is the primary way to assess ventricular function in children. Although there is extensive experience with this technology, data are limited on how ventricular function changes over time in children, which is a major impediment to conducting controlled trials of therapy in children.
This study has been approved by the Institutional Review Boards/Research Ethics Boards of all participating clinical centers:
Hospital for Sick Children, Toronto, Canada
Children's Hospital Boston, Boston, MA
Columbia College of Physicians and Surgeons, New York, NY
Children's Hospital of Philadelphia, Philadelphia, PA
Duke University Medical Center, Durham, NC
Brody School of Medicine at East Carolina University, Greenville, NC
Wake Forest Baptist Medical Center, Winston Salem, NC
Medical University of South Carolina, Charleston, SC
Primary Children's Medical Center, Salt Lake City, UT
Washington University, St. Louis, MO
DESIGN NARRATIVE:
This observational study will prospectively evaluate pediatric patients with dilated cardiomyopathy who are undergoing clinically indicated echocardiographic evaluation of LV function. Patients who undergo at least two echocardiograms 3 to 18 months apart will have their studies performed by the same ultrasonographer. All studies will be sent to the core laboratory for evaluation of variability in LV mass, volume, and ejection fraction.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients up to 22 years of age with dilated cardiomyopathy followed at participating clinical centers or newly diagnosed during the recruitment period
Key inclusion criteria (8 listed)
- Less than 22 years of age
- Any race
- Diagnosis of dilated cardiomyopathy
- LV and diastolic diameter greater than 5.5 cm (or z-score for BSA greater than 2) on the primary image acquisition from the first study echocardiogram
- LV ejection fraction less than 50% (or z-score for age less than -2) or shortening fraction less than 28% (or z-score for age less than -2) as measured on the primary image acquisition from the first study echocardiogram
Key exclusion criteria (12 listed)
- Hypertrophic cardiomyopathy
- Restrictive cardiomyopathy
- Myocardial noncompaction (LV hypertrabeculation); patient is eligible for the study as long as the echocardiogram performed at the time of screening has no evidence of myocardial noncompaction
- Ventricular paced rhythm
- Atrial or ventricular ectopy at ratio greater than 1:4
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
Inter-study variability of echo measurements; variance at a single point in time and variance of change in measurements over time
Time frame: Measured for up to 18 months
3 secondary outcomes
Relative magnitude of the various sources of variability in echocardiographic outcomes in order to optimize operational procedures that can minimize variance
Time frame: Measured for up to 18 months
Interstudy variability of echocardiographically-derived indices of LV systolic and diastolic function derived from m-mode, spectral Doppler, and tissue Doppler techniques used in pediatric patients with dilated cardiomyopathy
Time frame: Measured for up to 18 months
Relationship of clinical status, including treatment, to the interstudy variability and repeatability of echocardiographic measurements.
Time frame: Measured for up to 18 months
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 25 Jan 2013.
- Derived from this study: PubMed 29133477Selamet Tierney ES, Hollenbeck-Pringle D, Lee CK, Altmann K, Dunbar-Masterson C, Golding F, Lu M, Miller SG, Molina K, Natarajan S, Taylor CL, Trachtenberg F, Colan SD; Pediatric Heart Network Investigators. Reproducibility of Left Ventricular Dimension Versus Area Versus Volume Measurements in Pediatric Patients With Dilated Cardiomyopathy. Circ Cardiovasc Imaging. 2017 Nov;10(11):e006007. doi: 10.1161/CIRCIMAGING.116.006007.
- Derived from this study: PubMed 24265000Lee CK, Margossian R, Sleeper LA, Canter CE, Chen S, Tani LY, Shirali G, Szwast A, Tierney ES, Campbell MJ, Golding F, Wang Y, Altmann K, Colan SD; Pediatric Heart Network Investigators. Variability of M-mode versus two-dimensional echocardiography measurements in children with dilated cardiomyopathy. Pediatr Cardiol. 2014 Apr;35(4):658-67. doi: 10.1007/s00246-013-0835-9. Epub 2013 Nov 22.
- Derived from this study: PubMed 24132734Molina KM, Shrader P, Colan SD, Mital S, Margossian R, Sleeper LA, Shirali G, Barker P, Canter CE, Altmann K, Radojewski E, Tierney ES, Rychik J, Tani LY; Pediatric Heart Network Investigators. Predictors of disease progression in pediatric dilated cardiomyopathy. Circ Heart Fail. 2013 Nov;6(6):1214-22. doi: 10.1161/CIRCHEARTFAILURE.113.000125. Epub 2013 Oct 16.
- Derived from this study: PubMed 22677278Colan SD, Shirali G, Margossian R, Gallagher D, Altmann K, Canter C, Chen S, Golding F, Radojewski E, Camitta M, Carboni M, Rychik J, Stylianou M, Tani LY, Selamet Tierney ES, Wang Y, Sleeper LA; Pediatric Heart Network Investigators. The ventricular volume variability study of the Pediatric Heart Network: study design and impact of beat averaging and variable type on the reproducibility of echocardiographic measurements in children with chronic dilated cardiomyopathy. J Am Soc Echocardiogr. 2012 Aug;25(8):842-854.e6. doi: 10.1016/j.echo.2012.05.004. Epub 2012 Jun 5.
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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.
Lynn Sleeper, ScD principal investigator
Carelon Research
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cardiomyopathy, Dilated, run by Carelon Research at 8 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 8 inclusion and 12 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 25 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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?
Inter-study variability of echo measurements; variance at a single point in time and variance of change in measurements over time (time frame: Measured for up to 18 months). 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 NCT00123071, which also lists 4 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 25 Jan 2013.