Natural History Study in Patients With LGMDR5/2c

A Prospective Longitudinal Study of Natural History and Functional Status of Patients With Gamma-sarcoglycanopathy (LGMDR5)

NCT06210672•Atamyo Therapeutics

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Summary

Prospective, longitudinal, interventional, multicenter natural history study to better understand the LGMDR5/2c disease physiopathology. The duration of participation for each patient will be up to 24 months.

Detailed Description

Study duration Duration from First visit of first patient (FPFV) to Last visit of last patient (LPLV) : 3 years. Each patient will have a visit every 6 months over 2 years (5 visits in total). Study primary objective is to characterize the disease course in gamma-sarcoglycanopathy (LGMDr5/2c) patients using standardized and disease appropriate evaluations. Secondary objectives are to identify clinical, imaging and/or laboratory parameters that are indicators of the disease course in LGMD2c and to identify the best outcome measure for further therapeutics approaches

Eligibility

Age

6 - 35 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Male and female patients 6 to less than 35 years of age
•Ambulant patients should be less than 12 years of age and able to walk/run 10 meters (item 29 of NSAD) in less than 15 sec
•Confirmed diagnosis of LGMDR5 (genotyping)
•FVC \> 40%

Exclusion Criteria

•Subjects with co-morbidity which may interfere with LGMDR5 natural evolution and/or evaluation of outcome measures
•Need of non-invasive ventilation \> 16h per 24h or any invasive ventilation
•Left ventricular ejection fraction (LVEF) \< 30% or prior heart failure decompensation requiring hospitalization
•Past participation in a gene therapy or cell therapy trial

Locations (3)

Hopital Raymond Poincare

Garches, France

Hedi Chaker Hospital Child Neurology Department

Sfax, Tunisia

National Institute Mongi Ben Hmida of Neurology

Tunis, Tunisia

Key Dates

Start Date

January 10, 2025

Primary Completion Date

September 1, 2025

Completion Date

September 1, 2025

Last Updated

October 6, 2025

Conditions

Gamma-sarcoglycanopathy

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 6, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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