Ignite Creation Date:
2024-10-26 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06565572
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
None
First Post:
2023-08-09
Brief Title:
Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Sponsor:
None
Organization:
None
Study Overview
Official Title:
An Open-label Single Center Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for PCARP Posterior Column Ataxia With Retinitis Pigmentosa Disease Due to Mutations in FLVCR1
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-08
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:
The goal of this clinical trial is to evaluate a specific antisense oligonucleotide medication in one patient with posterior column ataxia with retinitis pigmentosa The main question it aims to answer is what is the safety and tolerability of this medication in a single participant
Detailed Description:
None
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