Ignite Creation Date:
2024-07-17 @ 11:09 AM
Last Modification Date:
2024-10-26 @ 3:34 PM
Study NCT ID:
NCT06491927
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-07-09
First Post:
2024-07-01
Brief Title:
Long Term Follow-up for RGX-202
Sponsor:
REGENXBIO Inc
Organization:
REGENXBIO Inc
Study Overview
Official Title:
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy DMD
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-07
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:
RGX-202-5101 is a long-term follow up study that evaluates the long term safety and efficacy of RGX-202 in participants who have received RGX-202 a gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal CT domain in a separate parent study
Detailed Description:
This is a multicenter prospective observational study to evaluate the long-term safety and efficacy of RGX-202 Eligible participants are those who have undergone evaluation in a previous parent clinical study following a single intravenous infusion of RGX-202 for the treatment of DMD Enrollment in the current long-term follow-up LTFU study will occur after the participant has completed or discontinued from the parent study Participants will be followed in this study cumulatively for up to 5 years after RGX-202 administration inclusive of the parent study No investigational treatment will be administered under this protocol The total study duration for each participant may vary depending on when he enrolls in the current study following RGX-202 administration in the parent study
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