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Evaluation of Cardiac Affections in Patients With Mucopolysaccharidosis (MPS) in Assuit University Children Hospital (AUCH)

Sponsor
Assiut University (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT04637646
Collaborator
(none)
25
Enrollment
13.9
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

This study aims to evaluate the different cardiac changes in the various types of MPS disorder and define the outcome of these cardiac abnormalities in those patients admitted to assuit University Children Hospital

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    The mucopolysaccharidoses (MPSs) are inherited lysosomal storage disorders caused by the absence of functional enzymes that contribute to the degradation of glycosaminoglycans (GAGs). The progressive systemic deposition of GAGs results in multi-organ system dysfunction that varies with the particular GAG deposited and the specific enzyme mutation(s) present.

    Cardiac involvement has been reported in all MPS syndromes and is a common and early feature, particularly for those with MPS I, II, and VI. Cardiac valve thickening, dysfunction (more severe for left-sided than for right-sided valves), and hypertrophy are commonly present; conduction abnormalities, coronary artery and other vascular involvement may also occur. Cardiac disease emerges silently and contributes significantly to early mortality. The clinical examination of individuals with MPS is often difficult due to physical and, sometimes, intellectual patient limitations. The absence of precordial murmurs does not exclude the presence of cardiac disease. Echocardiography and electrocardiography are key diagnostic techniques for evaluation of valves, ventricular dimensions and function.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    25 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Evaluation of Cardiac Affections in Patients With Mucopolysaccharidosis (MPS) in Assuit University Children Hospital (AUCH)
    Anticipated Study Start Date :
    Dec 1, 2020
    Anticipated Primary Completion Date :
    Dec 29, 2021
    Anticipated Study Completion Date :
    Jan 29, 2022

    Arms and Interventions

    Arm Intervention/Treatment
    children with MPS

    Outcome Measures

    Primary Outcome Measures

    1. Rate of Cardiac affection in patients with MPS [on enrollment]

      cardiac affection in MPS patients

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Day to 18 Years
    Sexes Eligible for Study:
    All
    Inclusion Criteria:
    • All the patients at any age up to 18 years with any type of MPS confirmed by the enzymatic assay
    Exclusion Criteria:
    • any patient other than MPS

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Assiut University

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Hager Rageh Dardiry, researcher, Assiut University
    ClinicalTrials.gov Identifier:
    NCT04637646
    Other Study ID Numbers:
    • cardiac affections in MPS
    First Posted:
    Nov 20, 2020
    Last Update Posted:
    Nov 20, 2020
    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:
    Mucopolysaccharidoses

    Study Results

    No Results Posted as of Nov 20, 2020