Ignite Creation Date:
2025-12-24 @ 5:50 PM
Ignite Modification Date:
2025-12-29 @ 2:25 AM
Study NCT ID:
NCT07197268
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-09-29
First Post:
2025-07-24
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation
Sponsor:
n-Lorem Foundation
Organization:
Study Overview
Official Title:
An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Bainbridge-Ropers Syndrome Due to ASXL3 Gene Variant
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-07
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 research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3
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 participant with BRPS due to a pathogenic, de novo nonsense variant in ASXL3
Study Oversight
Has Oversight DMC:
True
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?: