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

NCT ID: NCT05330039

Last Updated: 2024-03-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

22 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-11-30

Study Completion Date

2024-02-27

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Inborn Errors of Metabolism Propionic Aciduria Methylmalonic Aciduria Urea Cycle Disorder

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Patients with IEM

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

No interventions assigned to this group

Healthy Siblings

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

No interventions assigned to this group

Eligibility Criteria

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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
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University Children's Hospital

OTHER

Sponsor Role collaborator

Emma Marie Caroline Slack

OTHER

Sponsor Role lead

Responsible Party

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Emma Marie Caroline Slack

Prof. Dr.

Responsibility Role SPONSOR_INVESTIGATOR

Locations

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University Children's Hospital

Zurich, , Switzerland

Site Status

Countries

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Switzerland

Other Identifiers

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IEM

Identifier Type: -

Identifier Source: org_study_id

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