Trial record 9 of 98 for:    "DMD-associated dilated cardiomyopathy" OR "Cardiomyopathy, Dilated"

Pathophysiology of Dilated Cardiomyopathy

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified November 2013 by Great Ormond Street Hospital for Children NHS Foundation Trust
Sponsor:
Information provided by (Responsible Party):
Great Ormond Street Hospital for Children NHS Foundation Trust
ClinicalTrials.gov Identifier:
NCT02001961
First received: November 29, 2013
Last updated: NA
Last verified: November 2013
History: No changes posted
  Purpose

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
Dilated Cardiomyopathy

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Great Ormond Street Hospital for Children NHS Foundation Trust:

Primary Outcome Measures:
  • Fibrosis [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Higher fibrosis score (ECV) in heart failure patients in comparison to control subjects


Secondary Outcome Measures:
  • Biomarkers [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Significantly different pattern of blood biomarkers in heart failure patients as compared to control subjects

  • Disease Severity [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 20
Study Start Date: January 2014
Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts
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
Control subjects will be identified after being referred for an MRI scan and being allocated to a non-cardiac MRI with gadolinium contrast.

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   up to 16 Years
Genders Eligible for Study:   Both
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)
Criteria

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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02001961

Locations
United Kingdom
Great Ormond Street Hospital NHS Trust Not yet recruiting
London, United Kingdom, WC1N 3JH
Contact: Emma Pendleton       R&DGovernance@gosh.nhs.uk   
Principal Investigator: Dilveer K Panesar, Mb ChB         
Sponsors and Collaborators
Great Ormond Street Hospital for Children NHS Foundation Trust
Investigators
Study Director: Michael Burch, MD Great Ormond Street Hospital
  More Information

No publications provided

Responsible Party: Great Ormond Street Hospital for Children NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT02001961     History of Changes
Other Study ID Numbers: 12CC23
Study First Received: November 29, 2013
Last Updated: November 29, 2013
Health Authority: United Kingdom: Research Ethics Committee

Keywords provided by Great Ormond Street Hospital for Children NHS Foundation Trust:
Fibrosis
collagen metabolism
paediatric, dilated cardiomyopathy
biomarkers
T1 mapping
MRI

Additional relevant MeSH terms:
Cardiomyopathy, Dilated
Cardiomyopathies
Cardiomegaly
Heart Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on August 20, 2014