Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorVeonGen Therapeutics GmbH

About this trial

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.

Eligibility criteria

Qualifiers

Able to understand and willing to consent to study participation by a written informed consent

Male or female ≥ 18 years of age

Clinical diagnosis of RP

Confirmed pathogenic, biallelic variants in the CNGA1 gene

Disqualifiers

Additional interfering ocular conditions which would impact study results (e.g., ocular opacity and advanced cataract, uveitis, amblyopia)

History or presence of glaucoma

Ocular surgery, intravitreal or subretinal implantation of a medical device (within 6 months of screening)

Mutations known to cause inherited retinal disease other than biallelic variants in the CNGA1 gene

Trial design

Treatments tested in this trial

  • VG901

Treatment groups

6 Participants
are divided into 1 treatment group

Sponsors and collaborators