Ignite Creation Date:
2024-10-25 @ 8:05 PM
Last Modification Date:
2024-10-26 @ 3:39 PM
Study NCT ID:
NCT06588803
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-21
Brief Title:
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 Spinal Muscular Atrophy SMA
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a long term safety follow up study of patients in the EXG001-307-102 gene therapy clinical trial for SMA Type 1 Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 5 years
Detailed Description:
This study plans to enroll type 1 SMA patients who have previously received EXG001-307 treatment in the main trial EXG001-307-102 for long-term follow-up to observe the long-term safety and efficacy of the subjects The first year of long-term follow-up will be conducted every 3 months at the research center In the second year follow-up visits will be conducted at the center every six months with telephone follow ups at 15 and 21 months during the period From 2 years to 5 years after administration follow up by phone every six months and visit the center once a year
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