ProQR Therapeutics doses first patient in Phase 2/3 Illuminate sepofarsen trial

ProQR Therapeutics N.V. announced the first patient dosed in the Phase 2/3 ILLUMINATE clinical trial for sepofarsen in patients with Leber’s Congenital Amaurosis 10. Data from the study are expected at year-end 2020. “The start of the ILLUMINATE trial marks an important milestone towards our goal of bringing this novel and most advanced therapy for LCA10 to patients,” commented Dr. Aniz Girach, Ophthalmologist and Chief Medical Officer of ProQR. “With the ongoing trials in LCA10 and Ushers syndrome type 2, and the start of our clinical program for adRP later this year, we are breaking new ground and paving the way for new treatments in multiple severe inherited retinal diseases.” ILLUMINATE, or PQ-110-003, is a randomized, prospective, double-masked, sham-controlled 24-month trial of sepofarsen that 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 and a baseline best corrected visual acuity of 3.0 LogMAR or better. The trial is designed with the potential to serve as the sole registration trial for the program. Participants will be assigned equally to three parallel study arms; and a sham control arm with ten participants in each arm. Participants will receive a dose of sepofarsen or a sham-procedure at the start of the trial, at three months and then every six months afterwards. The primary endpoint will be mean change in BCVA from baseline in the active treated arms compared to the control arm.

https://thefly.com/landingPageNews.php?id=2892365