Ignite Creation Date:
2024-05-06 @ 12:45 PM
Last Modification Date:
2024-10-26 @ 1:03 PM
Study NCT ID:
NCT03842878
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-04-06
First Post:
2019-02-13
Brief Title:
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
Sponsor:
Genethon
Organization:
Genethon
Study Overview
Official Title:
Prospective Longitudinal Study of the Natural History and Functional Status of Patients With Limb-Girdle Muscular Dystrophy 2I
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Prospective longitudinal interventional single-group multicenter natural history study to better know the LGMD2I 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
Study objectives Primary objective
To characterize the disease course in Limb-Girdle Muscular Dystrophy 2I LGMD2I patients using standardized and disease appropriate evaluations
Secondary objectives
To identify clinical imaging andor laboratory parameters that are indicators of the disease course in LGMD2I To identify the best outcome measure for further therapeutics approaches
Study objectives Primary objective
To characterize the disease course in Limb-Girdle Muscular Dystrophy 2I LGMD2I patients using standardized and disease appropriate evaluations
Secondary objectives
To identify clinical imaging andor laboratory parameters that are indicators of the disease course in LGMD2I To identify the best outcome measure for further therapeutics approaches
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None