Gene therapy for X-linked retinoschisis
Part of Eyes & vision clinical trials.
This trial tests a gene therapy called ATSN-201 for people with X-linked retinoschisis (XLRS), a condition that affects the retina and can cause vision loss. The therapy aims to fix the faulty gene that causes the condition, potentially halting vision loss and improving vision. Participants receive a one-time injection into the eye, and the study monitors safety and effectiveness.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be at least 5 years old (for the main study) or at least 6 years old for certain groups
- Have a clinical diagnosis of X-linked retinoschisis caused by changes in the RS1 gene. Males need one change; females need two.
- Have vision in at least one eye that is between 20/200 and 20/40 (corrected by glasses or contacts)
- Have a specific pattern of retinal splitting (called schisis) seen on a scan in that eye
- Not have had any eye surgery (including laser) in the study eye within the past 6 months, and not plan any during the trial
- Not have any other eye condition that could increase risk of vision loss from the injection
- Not have had prior gene or cell therapy for the eye
- Not have used carbonic anhydrase inhibitors (medications for eye pressure or swelling) within the past month
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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