An observational study in Inborn Errors of Metabolism, Propionic Aciduria and Methylmalonic Aciduria, sponsored by Emma Marie Caroline Slack. Completed at 1 site in Switzerland. Open to participants aged Up to 18 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2024-03-01.
Sponsored by Emma Marie Caroline Slack · Observational
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.
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.
152 studies on the registry are indexed under Metabolism, Inborn Errors; 22 are open to participants now.
This study's enrollment of 22 is below the median of 65 across 45 observational studies indexed under Metabolism, Inborn Errors.
Browse Metabolism, Inborn Errors studies →Emma Marie Caroline Slack is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
The study aims to enroll 40 children that suffer from IEM (UCD, PA, MMA) and their siblings over the course of approximately two years.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Collection of biological samples (stool and urine) and health-related data at two timepoints, three to six months apart.
Collection of biological samples (stool) and health-related data at two timepoints, three to six months apart.
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
Time frame: 6 months
Disease aggravating microbiota (microbiota target)
Identify members of the intestinal microbiota (species/strains) that are associated with IEM via differential abundance testing (microbiota target)
Time frame: 6 months
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Feb 2024. You cannot join it, but the record below documents what was studied.
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Emma Marie Caroline Slack