Clinical trial recruiting patients with Leber’s congenital amaurosis 10 (LCA10)

ProQR are conducting a Phase 2/3 clinical trial called Illuminate to measure the safety and effectiveness of an RNA therapy called sepofarsen in patients with LCA 10, specifically those with mutations in the CEP290 gene. Sepofarsen is an investigational drug that is designed to repair a defect in the RNA and produce a normal functioning CEP290 protein. As a result, this has the potential to stop the progression of disease or reverse some vision loss.

ProQR are a Dutch biotechnology company developing RNA therapies for genetic retinal disease. Their research and development pipeline includes programmes focusing on specific forms of Usher syndromeLeber congenital amaurosis (LCA), Stargardt disease and autosomal dominant retinitis pigmentosa (RP).

Illuminate is in fact a continuation of a Phase 1/2 clinical trial whereby promising safety results and vision improvement was observed in study participants. The Illuminate trial now aims to find out if different dosing regimens (amounts) of the drug is safe and results in improvements in vision (efficiency).

Initially 30 adults and children (eight years and older) will be included in Illuminate. During the study, patients will be assigned equally to three parallel study groups with 10 participants in each group. Two groups will receive different doses (amounts) of sepofarsen through intravitreal (into the eye) injections. The third group will serve as a control group and will undergo a sham-procedure (simulated intravitreal procedure but no study drug administered).

This study is ongoing in a number of clinical centers in North America and Europe, involving 30 patients over a 25 month period.

More information about this and other clinical trials underway by ProQR can be found on their website.

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