Ignite Creation Date:
2024-05-06 @ 2:56 PM
Last Modification Date:
2024-10-26 @ 1:40 PM
Study NCT ID:
NCT04475926
Status:
RECRUITING
Last Update Posted:
2024-03-28
First Post:
2020-07-14
Brief Title:
A Study of the Natural History of Participants With LGMD2ER4 LGMD2DR3 LGMD2CR5 and LGMD2AR1 4 Years of Age Who Are Managed in Routine Clinical Practice
Sponsor:
Sarepta Therapeutics Inc
Organization:
Sarepta Therapeutics Inc
Study Overview
Official Title:
Journey A Global Multicenter Longitudinal Study of the Natural History of Subjects With Limb Girdle Muscular Dystrophy LGMD Type 2E LGMD2ER4 Type 2D LGMD2DR3 Type 2C LGMD2CR5 and Type 2A LGMD2AR1
Status:
RECRUITING
Status Verified Date:
2024-10
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:
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E LGMD2ER4 Limb-girdle muscular dystrophy type 2D LGMD2DR3 Limb-girdle muscular dystrophy type 2C LGMD2CR5 or Limb-girdle muscular dystrophy type 2A LGMD2AR1
These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 5 years after enrollment for participants with LGMD2CR5 LGMD2DR3 and LGMD2ER4 with a North Star Assessment for Dysferlinopathy NSAD 25 at Baseline up to 3 years for participants with LGMD2CR5 LGMD2DR3 and LGMD2ER4 with a NSAD 25 at Baseline and up to 3 years for participants with LGMD2AR1 Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present
These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 5 years after enrollment for participants with LGMD2CR5 LGMD2DR3 and LGMD2ER4 with a North Star Assessment for Dysferlinopathy NSAD 25 at Baseline up to 3 years for participants with LGMD2CR5 LGMD2DR3 and LGMD2ER4 with a NSAD 25 at Baseline and up to 3 years for participants with LGMD2AR1 Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None