QR-110 Clinical trial: Study 001
Phase 1/2 “Safety and efficacy trial”
The international Phase 1/2 trial has been completed and top-line results are available. The trial was an open-label, safety, tolerability and efficacy trial of sepofarsen (QR-110) in Leber’s congenital amaurosis 10 (LCA10) patients due to one or two copies (compound heterozygous or homozygous respectively) of the p.Cys998X in the CEP290 gene (also known as the c.2991+1655A>G mutation). Sepofarsen is an investigational drug that is designed to be administered by injections into the vitreous of the eye (intravitreal injections).
Patients participating in the trial received intravitreal injections of sepofarsen at different dose levels into one eye during one year (patients received 1-4 injections in total). Both adults (at least 18 years old) and children (6-17 years old) participated in this study.
The trial was conducted at three specialized centers with significant expertise in genetic retinal disease: the University of Iowa, Iowa City, Iowa, U.S., the Scheie Eye Institute at the University of Pennsylvania, Philadelphia, U.S., and the Ghent University Hospital, Ghent, Belgium.
The primary objective of the trial was to test whether different dose levels of sepofarsen were safe and tolerated. Secondary objectives were to test for the restoration/improvement of vision (efficacy) and retinal structure measured through specialized ophthalmic tests, and the sepofarsen pharmacokinetics. Changes in quality of life were also being evaluated.