Status:
WITHDRAWN
Natural History Study in Patients With LGMDR5/2c
Lead Sponsor:
Atamyo Therapeutics
Conditions:
Gamma-sarcoglycanopathy
Eligibility:
All Genders
6-35 years
Brief 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...
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 ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 10 2025
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 1 2025
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT06210672
Start Date
January 10 2025
End Date
September 1 2025
Last Update
October 6 2025
Active Locations (3)
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1
Hopital Raymond Poincare
Garches, France, 92380
2
Hedi Chaker Hospital Child Neurology Department
Sfax, Tunisia
3
National Institute Mongi Ben Hmida of Neurology
Tunis, Tunisia