Ignite Creation Date:
2025-12-24 @ 10:41 PM
Ignite Modification Date:
2026-01-05 @ 1:18 PM
Study NCT ID:
NCT06291935
Status:
RECRUITING
Last Update Posted:
2025-03-28
First Post:
2024-02-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
Sponsor:
VeonGen Therapeutics GmbH
Organization:
Study Overview
Official Title:
A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations
Status:
RECRUITING
Status Verified Date:
2025-03
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:
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are:
* What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration?
* Are there any early signs of efficacy on visual function?
Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.
* What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration?
* Are there any early signs of efficacy on visual function?
Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
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?:
False
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| EU CT: 2023-504383-42-00 | OTHER | EMA | View |