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International Consortium for Multimodality Phenotyping in Adults With Non-compaction (NONCOMPACT)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04424030
Recruitment Status : Not yet recruiting
First Posted : June 9, 2020
Last Update Posted : June 9, 2020
Sponsor:
Collaborators:
The Cleveland Clinic
University of Pennsylvania
Erasmus Medical Center
Seoul National University Hospital
Information provided by (Responsible Party):
Koen Nieman, Stanford University

Brief Summary:
Non-compaction cardiomyopathy (NCCM) is a heterogeneous, poorly understood disorder characterized by a prominent inner layer of loose myocardial tissue, and associated with heart failure, stroke, severe rhythm irregularities and death. For a growing population diagnosed with NCCM there is a need for better risk stratification to appropriately allocate (or safely withhold) these impactful preventive measures. The goal of this international consortium is to improve care of patients with non-compaction cardiomyopathy. We hypothesize that comprehensive analysis of clinical, genetic, structural and functional information will improve risk stratification. In addition, we hypothesize that detailed structural analysis will allow for differentiation of pathological and benign patterns of non-compaction. In a large cohort of adult patients with suspected NCCM we will perform in-depth phenotyping, including clinical information, pedigree data, genetics, echocardiography and MRI, and follow patients for up to 3 years. We will apply machine-learning based analytics to develop predictive models and compare their performance to currently used models and treatment criteria. Secondly, in a subset of patients we will perform high-resolution cardiac CT for detailed structural characterization of the myocardial wall. We will investigate associations between myocardial structure and regional contractile function, as assessed by echo and MRI. The aim of this proposal is to identify a structural signature associated with pathological non-compaction and improve developed risk prediction models. Discovery of pathological structural signatures through innovative imaging techniques, in relation to myocardial contractility, will advance our understanding of NCCM.

Condition or disease Intervention/treatment
Non-Compaction Cardiomyopathy Diagnostic Test: Echocardiography Diagnostic Test: Magnetic resonance imaging (MRI) Diagnostic Test: Computed tomography (CT)

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Study Type : Observational
Estimated Enrollment : 600 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: International Consortium for Multimodality Phenotyping in Adults With Non-compaction
Estimated Study Start Date : July 1, 2020
Estimated Primary Completion Date : April 1, 2025
Estimated Study Completion Date : April 1, 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cardiomyopathy

Group/Cohort Intervention/treatment
Multimodality imaging
Patients who have undergone echocardiography and cardiac MRI as part of their clinical management A research cardiac CT scan will be performed in eligible participants
Diagnostic Test: Echocardiography
Standard echocardiography exam performed as part of clinical management.

Diagnostic Test: Magnetic resonance imaging (MRI)
Standard comprehensive cardiac MRI exam of the heart performed as part of clinical management.

Diagnostic Test: Computed tomography (CT)
ECG-triggered, contrast-enhanced CT scan of the heart performed for research purposes in eligible study participants.




Primary Outcome Measures :
  1. Incidence of hard embolic adverse events [ Time Frame: Up to 4 years after enrollment ]
    Clinical neuro/systemic embolic event by autopsy, imaging or specialist evaluation

  2. Incidence of hard arrhythmic adverse events [ Time Frame: Up to 4 years after enrollment ]
    Sudden death (aborted), appropriate ICD discharge or VT/VF on ECG or rhythm/device monitoring


Secondary Outcome Measures :
  1. Incidence of hard heart failure related adverse events [ Time Frame: Up to 4 years after enrollment ]
    Heart failure death, cardiac transplantation or mechanical circulatory support

  2. Incidence of composite of hard adverse events [ Time Frame: Up to 4 years after enrollment ]
    Hard embolic, arrhythmic and heart failure related adverse events (as described above)

  3. Incidence of all embolic adverse events [ Time Frame: Up to 4 years after enrollment ]
    Hard embolic adverse events or transient neurologic event without objective infarction

  4. Incidence of all arrhythmic adverse events [ Time Frame: Up to 4 years after enrollment ]
    Hard arrhythmic adverse events or syncope without recorded arrhythmia

  5. Incidence of all heart failure related adverse events [ Time Frame: Up to 4 years after enrollment ]
    Hard heart failure related adverse events or resynchronization therapy, heart failure hospital admission

  6. Incidence of composite of adverse events [ Time Frame: Up to 4 years after enrollment ]
    All embolic, arrhythmic or heart failure related adverse events (as described above)


Biospecimen Retention:   Samples With DNA
After enrollment, blood will be drawn via venipuncture. The blood samples (35cc in total) will be centrifuged (15 minutes at 3500 RPM), after which the plasma, serum, red cells and buffy coat will be aliquoted from the vacutainers into color-coded vials. In addition to standard EDTA tubes we may include a CPT or PAX tube for more extensive analytics, or an Oragene tube for remote saliva collection. The aliquots and tubes will be stored at -70⁰C. The samples will remain at the site until analysis.


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with hypertrabeculation of the left ventricle fulfilling echo-based criteria of non-compaction cardiomyopathy
Criteria

Inclusion Criteria:

  • ≥18 years old
  • Hypertrabeculation of the left ventricle fulfilling the echo-based Jenni criteria of NCCM
  • Clinical cardiac MRI examination performed or planned

Exclusion Criteria (general cohort):

  • Complex congenital disease (including transposition great arteries, tetralogy of Fallot, tricuspid atresia, truncus arteriosis, single ventricle, hypoplastic left heart, pulmonary atresia, double-outlet RV), neuromuscular disorders or isolated RV non-compaction
  • Inability to provide informed consent
  • Contra-indications to MRI, which apply if the clinical cardiac MRI has not yet been performed at the time of study enrollment: permanent pacemakers/ICDs, MRI contrast medium allergy, significant arrhythmia with highly irregular RR intervals, severe dyspnea with inability to lay flat/breath hold, inability to communicate with the MRI technician or follow commands for any reason (psychosis, agitation, etc.), other site-specific contra-indications to clinical MRI of the heart.

Exclusion Criteria (cardiac CT examination):

  • Age <21 years
  • Decompensated heart failure, or otherwise clinically unstable
  • BMI>40 kg/m2
  • Pregnancy (or cannot be ruled out)
  • Known iodine contrast medium allergy
  • Kidney dysfunction: eGFR<45 ml/min
  • Thyroid disease: toxic multinodular goiter, Graves' disease, Hashimoto's thyroiditis

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 ClinicalTrials.gov identifier (NCT number): NCT04424030


Contacts
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Contact: Koen Nieman, MD, PhD 6507237476 knieman@stanford.edu
Contact: Nisha Udupa, BS 6507364363 nishaudupa@stanford.edu

Sponsors and Collaborators
Stanford University
The Cleveland Clinic
University of Pennsylvania
Erasmus Medical Center
Seoul National University Hospital
Investigators
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Principal Investigator: Koen Nieman, MD, PhD Stanford University
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Responsible Party: Koen Nieman, Associate Professor of Medicine, Stanford University
ClinicalTrials.gov Identifier: NCT04424030    
Other Study ID Numbers: 56001
SSU00099737 ( Other Identifier: Advarra Single IRB )
First Posted: June 9, 2020    Key Record Dates
Last Update Posted: June 9, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified datasets will be created and maintained on a public website in a password-protected area, where the frequency of use of the datasets can be monitored by Stanford and NHLBI.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Koen Nieman, Stanford University:
Echocardiography
Magnetic resonance imaging
Computed tomography
Prognosis
Genetics
Additional relevant MeSH terms:
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Cardiomyopathies
Heart Diseases
Cardiovascular Diseases