Observatory for patients with Type 3 Glycogenosis
Official title: French Observatory for Patients with Type 3 Glycogenosis
Run by Institut de Myologie, France · NCT06616545 · Data updated 2026-11-12
This study is creating an online registry to gather information from people with Glycogen Storage Disease Type III to better understand their experiences and improve future treatments.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 150 (estimated) |
| Sponsor | Institut de Myologie, France Academic / other |
| Locations | 3 sites (Clamart and 2 other locations) |
| Status | Recruiting |
| Started | September 1, 2013 |
| Main results expected | December 2026 |
| Study ends | December 2026 |
| Registry record updated | September 27, 2024 |
| Trial ID | NCT06616545 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are patients with molecularly characterized Glycogen Storage Disease Type III.
✕Probably not a fit
Patients diagnosed with GSD type 3 who refuse to participate in the study may not receive any benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study focuses on patients with Glycogen Storage Disease Type III, a genetic disorder caused by mutations in the AGL gene that affects glycogen metabolism. The study aims to create a multicenter online registry to collect data on patients' clinical characteristics, symptoms, and management strategies. By documenting the experiences of these patients, the registry seeks to improve understanding of the disease and inform future treatment approaches. Currently, the only management available is dietary intervention to limit glycogen storage and prevent hypoglycemia.
Why it matters
Potential benefit: If successful, this registry could enhance the understanding of Type 3 Glycogenosis and lead to improved management strategies for patients.
How similar studies have gone: While this approach is observational and may not have direct treatment implications, similar registries have successfully improved understanding and management of rare diseases.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients with molecularly characterised Glycogen Storage Disease Type III Exclusion Criteria: * Patients diagnosed with GSD type 3 refusing to take part in the study
Where it's running
-
Aphp Antoine Beclere
Clamart, France Recruiting -
CHU du Kremlin-Bicêtre
Le Kremlin-Bicêtre, France Recruiting -
Institue of Myology
Paris, France Recruiting
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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.