Trials / NCT06291935
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
Recruiting now Phase 1 Gene therapy
- Sponsor
- VeonGen Therapeutics GmbH
- Treatment
- VG901
- Gene
- CNGA1
- Conditions
- Retinitis Pigmentosa
- Ages
- 18 Years to no upper limit
- Enrollment
- 6 participants
- Started
- 2023-09-01
- Main results expected
- 2026-04
- Results posted
- Not yet
- Record last updated
- 2025-03-28
What the study is testing
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.
Where it runs
Germany (1)
- Center for Ophthalmology, University of Tuebingen, Tübingen (recruiting now)