ProQR Therapeutics has begun dosing in a phase 2/3 trial of sepofarsen, its treatment candidate for Leber’s congenital amaurosis 10, according to a press release.
The randomized, prospective, double-masked, sham-controlled 24-month ILLUMINATE trial will initially enroll 30 adults and children who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene.
There will be three parallel study arms: 40 µg (with an 80-µg loading dose), 80 µg (with a 160-µg loading dose) and sham control.
Sepofarsen, formerly named QR-110, is an RNA-based oligonucleotide designed to address the underlying causes of LCA10. The study’s primary endpoint will be mean change in best corrected visual acuity from baseline in the active treated vs. control arms, according to the release.