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
Uncategorized