VISTA - AGTC-RPGR-002 (X-Linked Retinitis Pigmentosa) - Clinical Trial
What is the Purpose of this Study?
We are doing this study to find out if an experimental drug called AGTC-501 (the study drug) can preserve and/or improve the vision of people who have X-linked retinitis pigmentosa (XLRP). We also want to know what dose of the drug has the most desirable effects.
X-linked Retinitis Pigmentosa (XLRP)
Who Can Participate in the Study?
Male children and adults ages 12-50 who:
- Are diagnosed with XLRP
- Do not have any artificial retinal implant or prosthesis
For more information, contact the study team at DEC-RA@duke.edu.
What is Involved?
If you choose to join this study and are eligible, you will get a random assignment (like drawing a number from a hat) to:
- Get a high dose of the study drug; OR
- Get a low dose of the study drug; OR
- Be in the control group (you will not receive the study drug in this group)
If you are is assigned to the high-dose or low-dose groups, you will have eye surgery to inject the study drug into one of your eyes (called the "study eye").
If you are assigned to the low dose or the high dose groups, you will participate for a total of 5 years. You will have at least 15 study visits over the first two years of the study. After your 24-month visit, long-term follow-up visits will occur at Year 3, Year 4, and Year 5. We will ask you to participate in the study for the entire 5 years.
If you are assigned to the control group, you will participate in the study for at least one year without receiving the study drug to help us learn more about XLRP. After at least one year, you will be reassessed for the opportunity to receive the high dose of the study drug in one of your eyes. If you choose to get the study drug atter your assessment, you will participate for a total of 6 years with about 23 visits. If you do not receive study drug, your participation in the study will end after 24 months and 10 visits.
Study Details
