ATSN-201-1 (LIGHTHOUSE) - Clinical Trial
What is the Purpose of this Study?
If you join this study, you will have a procedure where the study medicine is placed into the back of your eye. After this, you will return for follow up visits for up to five years so doctors can check how your eyes are doing. There will be about seventeen visits in total. At these visits, doctors will test your vision and examine your eyes to watch for changes and make sure the treatment is safe.
This study is for people who have a rare eye condition called X linked retinoschisis. This condition affects the back of the eye and can cause vision problems. It happens because of a change in a gene called RS1. Researchers want to learn more about this condition and how it affects vision.
Who Can Participate in the Study?
People can join this study if they are older than 6 years and have a diagnosis of X linked retinoschisis, which is a condition that affects vision. They cannot have taken part in another study that used gene therapy or cell therapy for the eyes before.
What is Involved?
This study is testing a new treatment called ATSN 201 for people who have a rare eye condition called X linked retinoschisis. This condition affects vision because of a change in a gene. The study drug is a type of gene therapy, which is designed to fix or replace the problem gene. Researchers want to learn if this treatment is safe and if it can help improve vision or slow down vision loss.
Study Details
