Non-invasive liver fibrosis testing in children and young adults with type III glycogen storage disease
Official title: Non-invasive Assessment of Liver Fibrosis in a French Cohort of Pediatric Patients With Type III Glycogen Storage Disease: Current State and Perspectives
Run by University Hospital, Strasbourg, France · NCT07303140 · Data updated 2026-11-12
This project tries to see if non-invasive liver stiffness measurements (like elastography) can monitor liver fibrosis in children and young adults with type III glycogen storage disease.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 1 Month to 21 Years |
| Sex | All |
| Enrollment | 30 (estimated) |
| Sponsor | University Hospital, Strasbourg, France Academic / other |
| Locations | 1 site (Strasbourg) |
| Status | Recruiting |
| Started | October 18, 2024 |
| Main results expected | February 2026 |
| Study ends | February 2026 |
| Registry record updated | December 24, 2025 |
| Trial ID | NCT07303140 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Children and young adults from birth up to 21 years with a confirmed diagnosis of type III glycogen storage disease who have undergone at least one liver elastography during follow-up are ideal candidates.
✕Probably not a fit
Patients who have never had liver elastography during follow-up, adults older than 21, or those without GSDIII are unlikely to benefit from this cohort analysis.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is an observational review of a French cohort of pediatric and young adult patients with type III glycogen storage disease who have had at least one liver elastography measurement. Investigators will collect elastography values along with clinical data, laboratory results, imaging, and liver biopsy findings when available to characterize fibrosis severity and progression. Analyses will describe relationships between elastography measurements and clinical outcomes such as liver failure, biopsy-proven fibrosis, or hepatocellular carcinoma. The goal is to summarize current practice and inform monitoring recommendations for this rare pediatric population.
Why it matters
Potential benefit: If successful, this work could support wider use of non-invasive elastography to monitor fibrosis and reduce the need for liver biopsies in these patients.
How similar studies have gone: Elastography has been validated for monitoring fibrosis in many adult liver diseases and is increasingly used in pediatric chronic liver conditions, but focused data specifically in pediatric GSDIII cohorts are limited.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Minors: from birth to 17 years * Adults: 18 to 21 years * Subjects being monitored for type III glycogen storage disease and having had at least one liver elastography measurement during their follow-up. Exclusion Criteria: \- Patients monitored for type III glycogen storage disease but who had never undergone liver elastography during their follow-up.
Where it's running
-
Service de pédiatrie 1 - CHU de Strasbourg - France
Strasbourg, France Recruiting
Study contacts
- Study coordinator: Camille WICKER, MD
- Email: camille.wicker@chru-strasbourg.fr
- Phone: 33 3 88 12 77 12
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.