Ignite Creation Date:
2024-05-06 @ 8:11 PM
Last Modification Date:
2024-10-26 @ 3:22 PM
Study NCT ID:
NCT06288230
Status:
RECRUITING
Last Update Posted:
2024-03-01
First Post:
2024-02-18
Brief Title:
An Open Label Study of Gene Therapy Product Vesemnogene Lantuparvovec in Spinal Muscular Atrophy
Sponsor:
Lantu Biopharma
Organization:
Lantu Biopharma
Study Overview
Official Title:
Study of AAV-hSMN1 Vesemnogene Lantuparvovec Gene Therapy in Subjects with Progressive Spinal Muscular Atrophy
Status:
RECRUITING
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:
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients
Detailed Description:
Study duration per participant is approximately 25 months including an approximately 30-day screeningbaseline period an approximately 24-month study observation period including 1 treatment day and an approximately 24-month follow-up period
Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose and followed by visits at months 2 3 6 12 18 and 24 post infusion Unscheduled visits may occur if the investigator determines that they are necessary
Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose and followed by visits at months 2 3 6 12 18 and 24 post infusion Unscheduled visits may occur if the investigator determines that they are necessary
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