Brighten is a clinical study for children under 8 years old with Leber congenital amaurosis (LCA) due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene. Around 15 children will participate in the clinical trial.
The pediatric participants will receive sepofarsen through intravitreal (into the eye) injections at one of five different dose levels. The aim of the Brighten study is to find out whether injections with sepofarsen are safe and tolerated in children with LCA.
Participants receive the study medicine up to a maximum of four times in one eye, the other eye remains untreated as a control. The treatment will be given on day one, followed by a dose every six months in the study. The clinical trial is being conducted at several hospitals in Europe.