ProQR to begin phase 2/3 trial of treatment for Leber’s congenital amaurosis 10

ProQR Therapeutics and the FDA have reached an agreement regarding the design of the phase 2/3 ILLUMINATE trial of the company’s RNA-based oligonucleotide treatment candidate for Leber’s congenital amaurosis 10, according to a press release.

The double-masked, randomized, sham-controlled ILLUMINATE trial will enroll 30 adults and children in three parallel arms to study sepofarsen, which was previously known as QR-110.

Subjects in the active arms will receive 40 µg doses with an 80 µg loading dose or 80 µg doses with a 160 µg loading dose of sepofarsen. Those in the control arm will receive sham injections. Each subject will receive injections at the start of the trial, at 3 months and then every 6 months. The primary efficacy endpoint is a change in visual acuity from baseline to 12 months.

“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,” David Rodman, MD, ProQR executive vice president of research and development, said in the release. “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.”

The trial is expected to begin in the first half of 2019.