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

Inborn Errors of Metabolism Clinical Trial

— IEM  

Official title:

Enterobacteria Strain-level Characterization in Children With Inborn Errors of Metabolism (IEM)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05330039
• Other study ID #: IEM
• Status: Completed
• Start date: November 30, 2021
• Est. completion date: February 27, 2024

Study information

• Verified date: February 2024
• Source: ETH Zurich
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: February 27, 2024
• Est. primary completion date: February 27, 2024
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Inborn Errors of Metabolism
• Metabolism, Inborn Errors
• Propionic Acidemia
• Urea Cycle Disorder
• Urea Cycle Disorders, Inborn

Locations

Country	Name	City	State
Switzerland	University Children's Hospital	Zürich

Sponsors (2)

Lead Sponsor	Collaborator
Emma Marie Caroline Slack	University Children's Hospital

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Contribution of micriobiota to disease severity	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	6 months
Secondary	Disease aggravating microbiota (microbiota target)	Identify members of the intestinal microbiota (species/strains) that are associated with IEM via differential abundance testing (microbiota target)	6 months