Study of Sepofarsen for LCA type 10
Part of Brain & nervous system, Eyes & vision, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests an investigational RNA therapy called sepofarsen for people with Leber Congenital Amaurosis (LCA) type 10, a rare inherited eye disease that causes severe vision loss from birth. The goal is to see if it can slow or improve vision loss.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have Leber Congenital Amaurosis type 10 caused by a specific change (mutation) in the CEP290 gene.
- You need to be at least 6 years old. There is no upper age limit.
- Your vision in the better eye must be no better than 20/50 (about 20/60 or worse) but still measurable with a special eye chart. If you only see light but used to see better, you may also qualify.
- Your eye disease must affect both eyes in a similar way.
- You must have a healthy layer of cells (the outer nuclear layer) in the back of the eye, confirmed by a special scan.
- You must not have had any prior gene therapy, RNA therapy, or stem-cell therapy for any condition (including sepofarsen).
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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