Ignite Creation Date:
2024-05-06 @ 8:15 PM
Last Modification Date:
2024-10-26 @ 3:24 PM
Study NCT ID:
NCT06314490
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-03-18
First Post:
2024-03-01
Brief Title:
Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease SCN2A
Sponsor:
University of California San Diego
Organization:
University of California San Diego
Study Overview
Official Title:
Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease SCN2A
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-03
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 research project entails delivery of a personalized antisense oligonucleotide ASO drug designed for a single pediatric participant with SCN2A associated developmental epileptic encephalopathy
Detailed Description:
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide ASO treatment in a single pediatric participant with a de novo pathogenic gain of function SCN2A mutation associated with severe developmental epileptic encephalopathy
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