Non-invasive Assessment of Liver Fibrosis in a French Cohort of Pediatric Patients With Type III Glycogen Storage Disease: Current State and Perspectives
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Liver Fibrosis. Basic parameters: 1 months — 21 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
Patients with type III glycogen storage disease (GSDIII) can develop liver fibrosis, which can be complicated by liver failure or even hepatocellular carcinoma. Since the beginning of the 21st century, non-invasive techniques for assessing fibrosis, such as liver elastography, have been developed. These techniques often make it possible to avoid liver biopsies during patient follow-up and have already been validated in the management of several diseases in adults. These techniques are also beginning to be recommended for monitoring certain chronic liver diseases in children.
Primary outcome measures
- Liver elastography values [Time frame: 1 hour after consultation]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Study design
- Observational model
- Cohort
Study locations
France · 1 center
- Service de pédiatrie 1 - CHU de Strasbourg - France — Strasbourg
Identifiers
NCT: NCT07303140 · 9479