Ignite Creation Date:
2024-05-06 @ 7:02 PM
Last Modification Date:
2024-10-26 @ 2:59 PM
Study NCT ID:
NCT05876780
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-09-21
First Post:
2023-05-17
Brief Title:
A Gene Transfer Single Dose Study to Evaluate the Safety Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy Type 2ER4 Beta-Sarcoglycan β-SG Deficiency
Sponsor:
Sarepta Therapeutics Inc
Organization:
Sarepta Therapeutics Inc
Study Overview
Official Title:
A Multicenter Open-label Single-dose Systemic Gene Transfer Study to Evaluate the Safety Tolerability and Efficacy of SRP-9003 on Subjects With Limb Girdle Muscular Dystrophy Type 2ER4 β-Sarcoglycan Deficiency
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-09
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:
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy type 2ER4 LGMD2ER4 The study will include both ambulatory Cohort 1 and non-ambulatory Cohort 2 participants
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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