ProQR Therapeutics announced that it has reached agreement with the FDA on the design of a Phase 2/3 pivotal trial for QR-110, now renamed to sepofarsen, in patients with Leber’s congenital amaurosis 10, or LCA10 due to the p.Cys998X mutation in the CEP290 gene. LCA is the leading genetic cause of childhood blindness. The trial is expected to start in the first half of 2019. We anticipate top line results for base-case of 30 trial participants around year-end 2020. ILLUMINATE will be a randomized, double-masked, sham-controlled trial initially enrolling 30 adults and children assigned equally to three parallel arms with 10 participants in each arm. The trial incorporates adaptive sample size re-estimation and is designed with potential to serve as the sole registration trial for the program. Participants will receive a dose of sepofarsen or a sham-injection at the start of the trial, at three months and then every six months. The primary efficacy endpoint for the trial will be change in visual acuity from baseline in the treated arm compared to sham-treated control arm at the 12 month time point. Sham-treated participants may be offered cross-over to active treatment after 12 months, and all participants will continue to receive treatment for a total of 24 months after which they may be offered participation in an open label extension trial. Treatment of the second eye will be adaptively incorporated into the trial.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.