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Pathophysiology of Dilated Cardiomyopathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02001961
Recruitment Status : Unknown
Verified November 2013 by Great Ormond Street Hospital for Children NHS Foundation Trust.
Recruitment status was:  Not yet recruiting
First Posted : December 5, 2013
Last Update Posted : December 5, 2013
Information provided by (Responsible Party):
Great Ormond Street Hospital for Children NHS Foundation Trust

Brief Summary:

This will be a cross-sectional, observational study.

Null hypothesis:

There is no difference in the amount of extracellular volume (ECV or scarring) in the hearts of patients with heart failure as compared to control subjects.

Heart failure occurs when the heart muscle has become too weak to work properly. It is associated with an increase in the amount of connective tissue (collagen) which replaces dead heart muscle cells (scarring). Currently a biopsy of the muscle is the only way to measure the amount of scarring. This is invasive and rarely done in children. Because of this, it is difficult to measure the amount of scarring in a particular patient or disease process, which is important for improving our understanding and treatment of the disease.

Cardiac magnetic resonance imaging (MRI) is a non-invasive imaging tool which is routinely used to look at areas of local scarring in heart muscle. Because the scarring is so widespread in paediatric patients, we have not been able to use this method previously. Now new imaging techniques allow us to look at widespread scarring but these have not yet been validated in children.

We plan to use late gadolinium enhancement (T1 mapping) to measure the amount of scarring in patients with heart failure (we have evidence that their heart biopsies show increased amounts of scar tissue) and children having MRI scans for other reasons. We will use measures of function including echocardiography and 6 minute walk test to compare to the amount of scarring. This will help us to know whether the amount of scarring will be clinically useful.

We will look at the amount of various proteins in the blood of patients and control subjects which are related to the scarring and cell death processes. We already use blood tests to monitor heart failure and these tests may help us to refine our testing and improve timing of treatment (e.g. transplantation).

This study will help us to design further research in this field.

Condition or disease
Dilated Cardiomyopathy

Show Show detailed description

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Case-Control
Time Perspective: Cross-Sectional
Official Title: Using Novel Blood and Imaging Biomarkers to Better Understand the Pathophysiology of Paediatric Dilated Cardiomyopathy
Study Start Date : January 2014
Estimated Primary Completion Date : June 2014

Heart failure
Patients will be recruited from the heart failure clinic by their consultant. These will include patients who are due to have a MRI scan for clinical reasons and those who volunteer to participate. Voluntary subjects will be over 8 years.
Control subjects will be identified after being referred for an MRI scan and being allocated to a non-cardiac MRI with gadolinium contrast.

Primary Outcome Measures :
  1. Fibrosis [ Time Frame: 6 months ]
    Higher fibrosis score (ECV) in heart failure patients in comparison to control subjects

Secondary Outcome Measures :
  1. Biomarkers [ Time Frame: 6 months ]
    Significantly different pattern of blood biomarkers in heart failure patients as compared to control subjects

  2. Disease Severity [ Time Frame: 6 months ]
    Significant correlation between fibrosis score on MRI or biomarker profile and clinical parameters (including 6 minute walk test and disease severity).

Biospecimen Retention:   Samples Without DNA
Blood plasma (>400 UL) will be collected in heparinised tubes plasma will be extracted and the cell fraction discarded. Plasma will be stored at 80°C prior to transport on dry ice, via protected courier.

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
  1. Heart Failure Patients (MRI Clinically indicated)
  2. Heart Failure Patients (Voluntarily recruited from clinic)

    1. 8-16 years of age
    2. Non-GA only
  3. Control subjects (Clinically indicated brain MRI with contrast)

Inclusion Criteria:

  • Established diagnosis of DCM for 3 months
  • Ability to cooperate with MRI scan without general anaesthesia (volunteers over 8 years), or any age if cardiac MRi clinically indicated
  • Provides written, informed consent

Exclusion Criteria:

  • Patient exclusion criteria:
  • Estimated GFR <30mls/min
  • Contraindication to MRI (see appendix 1)
  • Chronic inflammation/ malignancy/ connective tissue disease
  • Structural congenital heart disease/ previous cardiac surgery

Control Group Exclusion Criteria:

  • History of heart failure or congenital heart disease
  • Contraindication to MRI (see below)
  • Chronic inflammation/ malignancy/ connective tissue disease
  • Previous malignancy

Exclusion criteria for MRI

  • Central nervous system aneurysm clips
  • Implanted neural stimulator
  • Implanted cardiac pacemaker or defibrillator
  • Cochlear implant
  • Ocular foreign body e.g. metal shavings
  • Other implanted medical devices e.g. drug infusion ports
  • Insulin pump
  • Metal shrapnel or bullet
  • Pregnant women (patients who are uncertain will be required to have a urinary or blood screening test)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02001961

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United Kingdom
Great Ormond Street Hospital NHS Trust
London, United Kingdom, WC1N 3JH
Contact: Emma Pendleton       R&   
Principal Investigator: Dilveer K Panesar, Mb ChB         
Sponsors and Collaborators
Great Ormond Street Hospital for Children NHS Foundation Trust
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Study Director: Michael Burch, MD Great Ormond Street Hospital
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Responsible Party: Great Ormond Street Hospital for Children NHS Foundation Trust Identifier: NCT02001961    
Other Study ID Numbers: 12CC23
First Posted: December 5, 2013    Key Record Dates
Last Update Posted: December 5, 2013
Last Verified: November 2013
Keywords provided by Great Ormond Street Hospital for Children NHS Foundation Trust:
collagen metabolism
paediatric, dilated cardiomyopathy
T1 mapping
Additional relevant MeSH terms:
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Cardiomyopathy, Dilated
Heart Diseases
Cardiovascular Diseases