A Dutch maker of of transformative RNA medicines for the treatment of severe genetic rare diseases, ProQR Therapeutics, has got the U.S. Food and Drug Administration (FDA) tips for a Phase 2/3 pivotal trial Illuminate for sepofarsen, in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene, the leading genetic cause of childhood blindness.
The trial of previously known as QR-110, should start in next 6 months, with 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 (two active dose levels and a sham control arm) with 10 participants in each arm.
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.
David Rodman, M.D., Executive Vice President of Research and Development of ProQR, said: “Our regulatory discussions have been very collaborative and resulted in agreement on major design elements that allow us to take the safest and most expeditious path to further develop this potentially transformative therapy for patients who suffer from severe vision loss. Importantly, we will extend the dose exploration to an even lower and less frequent dosing regimen and include both a regimen similar to that tested in the Phase 1/2 trial as well as a sham-treated group, conditions that will allow us to evaluate safety and efficacy in a design that satisfies regulatory requirements for a pivotal trial. We are also pleased to start the open-label extension trial for participants in the Phase 1/2 trial so that they can continue to receive sepofarsen and have potential for treatment of the untreated eye.”