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.
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
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
Sequential
VG901
DrugAdministered as specified in the treatment arm. Study Cohort 1 - Low dose; Study Cohort 2 - High dose Other Names: Gene Therapy (AAV2.NN-CNGA1)
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Number of Adverse Events (AEs) and Serious Adverse Events (SAEs). Ocular inflammation is defined as an adverse event of special interest (AESI). AESI follow the same reporting requirements as SAE.
Physical Examination
A complete physical examination will be done at Screening and at Baseline and then at Month 12. On the other timepoints during the 1-year active follow-up phase, symptom-directed physical examination will be conducted. Abnormal examination results will be recorded.
Pulse rate
Will be recorded in beats per minute.
Blood pressure
Systolic and diastolic blood pressure will be recorded in mmHg.
Body temperature
Will be recorded in °C.
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