NCT07303140

Brief Summary

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.

Trial Health

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Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Oct 2024

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 18, 2024

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

December 11, 2025

Completed
13 days until next milestone

First Posted

Study publicly available on registry

December 24, 2025

Completed
1 month until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2026

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2026

Completed
Last Updated

December 24, 2025

Status Verified

December 1, 2025

Enrollment Period

1.3 years

First QC Date

December 11, 2025

Last Update Submit

December 11, 2025

Conditions

Keywords

Liver fibrosisType III glycogen storage diseaseLiver fibrosis in pediatric patients

Outcome Measures

Primary Outcomes (1)

  • Liver elastography values

    Liver fibrosis is classified into four stages, from 0 to 4: * F0 corresponds to the absence of fibrosis * F4 corresponds to established cirrhosis.

    1 hour after consultation

Eligibility Criteria

Age1 Month - 21 Years
Sexall
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Patients under 22 years of age

You may qualify if:

  • 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.

You may not qualify if:

  • \- Patients monitored for type III glycogen storage disease but who had never undergone liver elastography during their follow-up.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Service de pédiatrie 1 - CHU de Strasbourg - France

Strasbourg, 67091, France

RECRUITING

MeSH Terms

Conditions

Liver CirrhosisGlycogen Storage Disease Type III

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesFibrosisPathologic ProcessesPathological Conditions, Signs and SymptomsGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 11, 2025

First Posted

December 24, 2025

Study Start

October 18, 2024

Primary Completion

February 1, 2026

Study Completion

February 1, 2026

Last Updated

December 24, 2025

Record last verified: 2025-12

Locations