PRO-OMICS: Ventricular Remodelling and Metabolomics in Pediatric Cardiomyopathies (PROGRESS-OMICS)

Sponsor
Bambino Gesù Hospital and Research Institute (Other)
Overall Status
Enrolling by invitation
CT.gov ID
NCT04670731
Collaborator
Parent Project, Italy (Other), Ministry of Health, Italy (Other)
100
1
15.9
6.3

Study Details

Study Description

Brief Summary

The pathogenesis of cardiomyopathies is complex and a simple approach cannot describe the whole picture. Different etiologies are reported in pediatric age and heart failure onset can lead to poor prognosis in term of need of heart transplantation and ventricular assist device implantation. Based on hypothesis that heart failure development is related to heart inability to meet metabolic demands of the body, our study will focus to evaluate cardiac metabolism as one of the most critical factors and the accompanying changes of metabolic and echocardiographic profiles at different stages of heart failure. The heart is a unique organ working continuously as a pump supplying blood to the body. To meet this requirement, the myocardium utilizes fatty acids to generate 70-90% of the adenosine triphospate, with the rest being produced by oxidation of glucose, lactate, ketone bodies, aminoacids. Utilization of fatty acids is reduced in the failing heart and there is a metabolic shift to generation of adenosine triphospate from glucose. In patients with advanced cardiomyopathies, the heart is unable to utilize either metabolite and thus "runs out of fuel". It is reported that the adenosine triphospate level is approximately 30% lower in failing human hearts compared with non-failing hearts. In addition to this premise about the metabolic profile of the failing heart, recent advances in the field of metabolomics have indicated that several metabolites and/or metabolic pathways have a role in heart failure. Metabolism of lipids, glycolysis, fructolysis, aminoacids, and ketone oxidation have been found to be altered in non-ischemic cardiomyopathy in adult population. Also in adult heart failure patients some metabolic profiles resulted pronounced perturbated. Taking advantage of the high throughput, metabolomics is a platform for identifying metabolic signatures in children at each stages of heart failure (from pre clinical heart failure to end stage forms). We also will determine whether metabolomic analysis provides sensitive evaluation of heart failure in terms of remodelling at different stages and in disease regression after therapeutic interventions. Study desing is conceived in two parts. The first part is retrospective and we will analyze all echocardiograms in all children affected by cardiomyopathies. The second part is a cross sectional study in which will evaluate untargeted metabolomics in children at any stage of heart failure (A,B, C, D) and in control group. We will evaluate the clinical applicability and significance of plasma metabolomic analysis in the diagnosis and prognosis of heart failure in pediatric ages.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Cohort
    Time Perspective:
    Cross-Sectional
    Official Title:
    Study of Ventricular Remodelling and Metabolomics in Pediatric Cardiomyopathies (PROGRESS-OMICS)
    Anticipated Study Start Date :
    Feb 1, 2021
    Anticipated Primary Completion Date :
    Jun 1, 2021
    Anticipated Study Completion Date :
    Jun 1, 2022

    Outcome Measures

    Primary Outcome Measures

    1. Identification of untargeted metabolomic profiles [1 year]

      Identification of metabolomic profiles in children with heart failure according to different stages od heart failure (A, predisposing condition; B- asymptomatic stage; C- symptomatic; D: end stage) and control group

    Secondary Outcome Measures

    1. metabolomic profile and ventricular remodelling [1 year]

      correlation between metabolomic profiles and echocardiographic characteristic, such as left ventricular mass, left ventricular dilatation, mass/volume ratio) according to different stages of heart failure (A, predisposing condition; B- asymptomatic stage; C- symptomatic; D: end stage)

    2. metabolomic profile and ventricular remodelling [1 year]

      correlation between metabolomic profiles and ECG characteristics, such as duration of PR, QRS, T wave change, according to different stages of heart failure (A, predisposing condition; B- asymptomatic stage; C- symptomatic; D: end stage)

    3. identification of potential diagnostic of metabolomic profile [1 year]

      correlation between metabolomic profiles and clinical outcome, such as clinical stage, progression of remolling, death, heart transplantion, acute hospitalization for heart failure, VAD implantation, heart transplantation

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Month to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    Yes

    Patients with Cardiomyopathies

    Inclusion Criteria:
    • Dilated Cardiomyopathy, defined as left ventricular dilation > 2SD

    • A-D stages of Heart Failure, according to ACC/AHA definition

    • < 18 years

    Exclusion Criteria:
    • Restrictive cardiomyopathy

    • Hypertrophic cardiomyopathy

    • Congenital Heart Diasease

    • Valvular Heart Disease, as primary cause of heart failure

    • 18 years

    Control Group

    Inclusion Criteria:
    • Children without familial disease and/ or abnormalities of ECG and echocardiographic, NT pro BNP ≥ 103 pg/mL, TnT ≥ 14
    Exclusion Criteria:
    • Children with familial disease and/or any abnormalities of ECG and echocardiographic abnormalities, NT pro BNP ≥ 103 pg/mL, TnT ≥ 14

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Bambino Gesù Hospital and Research Institute Rome Italy

    Sponsors and Collaborators

    • Bambino Gesù Hospital and Research Institute
    • Parent Project, Italy
    • Ministry of Health, Italy

    Investigators

    • Study Chair: Rachele Adorisio, MD, Bambino Gesù Hospital and Research Institute, Rome, Italy
    • Principal Investigator: Rachele Adorisio, MD, Bambino Gesù Hospital and Research Institute, Rome, Italy

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Bambino Gesù Hospital and Research Institute
    ClinicalTrials.gov Identifier:
    NCT04670731
    Other Study ID Numbers:
    • 2259
    First Posted:
    Dec 17, 2020
    Last Update Posted:
    Feb 10, 2021
    Last Verified:
    Nov 1, 2020
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Feb 10, 2021