Learn about the study

• The Vista trial is for males between the ages of 12 and 50 who have X-linked retinitis pigmentosa (XLRP) caused by a mutation (or defect) in the RPGR gene.
• The Vista trial is studying an investigational (not yet approved for sale by government authorities like the FDA in the US or EMA in Europe) new gene therapy called AGTC-501.
• The study drug (AGTC-501) is designed to provide a healthy version of the mutated (or damaged) RPGR gene to allow the cells in the retina to make a healthy RPGR protein.
• The purpose of the Vista trial is to assess how well 2 different doses of AGTC-501 might work to preserve and/or improve vision and other symptoms of XLRP when compared to study participants who have not received AGTC-501.
• The safety and tolerability of these 2 doses will also be assessed in this study.

How the Vista trial works

Qualification for the study involves confirmation of XLRP with an RPGR gene mutation by genetic testing and a review of your medical history to help determine if you or your child meet eligibility criteria.

• If eligibility criteria are met, participants will then attend 3 screening visits at a study site.
• Qualified participants will be randomly assigned to 1 of 2 treatment groups or the control group.
• Eligible participants in the treatment groups will undergo a procedure to receive an injection of AGTC-501 in one eye. The study doctor and study participants will not know which dose is given.
• Participants in the control group will not receive AGTC-501 initially, but they will have the option to receive AGTC-501 after 1 year if they meet eligibility requirements.
• Depending on group assignment, trial participation lasts from 2 to 6 years.