A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Summary

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment

Timeline

Start

2019-04-04

Primary completion

2022-01-31

Completion

2023-03

Outcome

Missed primary endpoint

release “Illuminate trial of sepofarsen for the treatment of CEP290 -mediated Leber congenital amaurosis 10 (LCA10) did not meet its primary endpoint” proqr.com ↗

Drugs

Indications