Observatory for patients with Type 3 Glycogenosis
French Observatory for Patients with Type 3 Glycogenosis
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 |
|---|---|
| Enrollment | 150 (estimated) |
| Sex | All |
| Sponsor | Institut de Myologie, France Academic / other |
| Locations | 3 sites (Clamart and 2 other locations) |
| Trial ID | NCT06616545 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are patients with molecularly characterized Glycogen Storage Disease Type III.
Not a fit: Patients diagnosed with GSD type 3 who refuse to participate in the study may not receive any benefit.
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 performed: While this approach is observational and may not have direct treatment implications, similar registries have successfully improved understanding and management of rare diseases.
Eligibility criteria
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 this trial is running
Clamart and 2 other locations
- Aphp Antoine Beclere — Clamart, France (Recruiting)
- CHU du Kremlin-Bicêtre — Le Kremlin-Bicêtre, France (Recruiting)
- Institue of Myology — Paris, France (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.