Ignite Creation Date:
2024-05-06 @ 8:22 AM
Last Modification Date:
2024-10-26 @ 11:59 AM
Study NCT ID:
NCT02725580
Status:
COMPLETED
Last Update Posted:
2023-03-08
First Post:
2016-03-16
Brief Title:
Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 vLINCL6 Disease
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
Phase IIIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis Delivering the CLN6 Gene by Self-Complementary AAV9
Status:
COMPLETED
Status Verified Date:
2023-03
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 a phase 12 open-label single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutations in the CLN6 gene vLINCL6 disease
Detailed Description:
This is an open-label single-dose study of AT-GTX-501 administered by a single intrathecal injection Safety and efficacy are evaluated over a 2 year period The efficacy assessments in this study are to evaluate motor language visual and cognitive function as well as survival and other outcome measures Participants are tested at baseline receive AT-GTX-501 on Day 0 and return for visits on Days 7 14 21 and 30 and then every 3 months until Month 24 Following completion of this study there is a long-term follow up study in which data will continue to be collected Study AT-GTX-501-02 NCT04273243
For more information about this study please contact Amicus Therapeutics Patient Advocacy at clinicaltrialsamicusrxcom or 1 609-662-2000
For more information about this study please contact Amicus Therapeutics Patient Advocacy at clinicaltrialsamicusrxcom or 1 609-662-2000
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