Clincosm LogoSign in Get a demo

Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)

Sponsor
Emma Marie Caroline Slack (Other)
Overall Status
Recruiting
CT.gov ID
NCT05330039
Collaborator
University Children's Hospital (Other)
80
Enrollment
1
Location
24
Anticipated Duration (Months)
3.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Study around children with inborn errors of metabolism (IEM) and their healthy siblings. Collection of stool and urine to assess contribution of microbiota to disease severity.

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    This study aims to collect biological samples (stool and urine) from children with inborn errors of metabolism, IEM (like UCD (urea cycle disorder), PA (propionic aciduria) and MMA (methylmalonic aciduria) and their healthy siblings.

    The main focus of the study is to assess the contribution of the intestinal microbiota to disease severity in children that suffer from different forms of IEM and potentially find microbiota targets that could be used in the design of therapeutic/prophylactic agents.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    80 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Enterobacteria Strain-level Characterization in Children With Inborn Errors of Metabolism (IEM)
    Actual Study Start Date :
    Nov 30, 2021
    Anticipated Primary Completion Date :
    Nov 30, 2023
    Anticipated Study Completion Date :
    Nov 30, 2023

    Arms and Interventions

    Arm Intervention/Treatment
    Patients with IEM

    Collection of biological samples (stool and urine) and health-related data at two timepoints, three to six months apart.
    Healthy Siblings

    Collection of biological samples (stool) and health-related data at two timepoints, three to six months apart.

    Outcome Measures

    Primary Outcome Measures

    1. Contribution of micriobiota to disease severity [6 months]

      Assess contribution of intestinal microbiota to disease severity in children that suffer from different forms of IEM via changes in frequencies, absolute colonization levels or strain identity of microbiota species

    Secondary Outcome Measures

    1. Disease aggravating microbiota (microbiota target) [6 months]

      Identify members of the intestinal microbiota (species/strains) that are associated with IEM via differential abundance testing (microbiota target)

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    Yes
    Inclusion Criteria:
    • Children or siblings of children that have been diagnosed with one of the following diseases: any type of UCD, PA or MMA
    Exclusion Criteria:

    • Children or siblings of children that have not been diagnosed with one of the following diseases: any type of UCD, PA or MMA

    • use of an investigational drug or device less than 30 days prior to the study, or current enrollment in another investigational drug or device study less than 30 days prior to the study

    • considered to be poor attendees or unlikely for any reason to be able to comply with the study procedures, in the opinion of the investigator

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University Children's Hospital Zürich Switzerland 8032

    Sponsors and Collaborators

    • Emma Marie Caroline Slack
    • University Children's Hospital

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Emma Marie Caroline Slack, Prof. Dr., ETH Zurich
    ClinicalTrials.gov Identifier:
    NCT05330039
    Other Study ID Numbers:
    • IEM
    First Posted:
    Apr 15, 2022
    Last Update Posted:
    Apr 15, 2022
    Last Verified:
    Apr 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Urea Cycle Disorders, Inborn
    Metabolism, Inborn Errors
    Propionic Acidemia

    Study Results

    No Results Posted as of Apr 15, 2022