PSS3 Topline Patient-Reported Outcome Results from a Multicenter, Open-Label, Phase 4 Study of Repository Corticotropin Injection in Patients with Refractory Severe Noninfectious Keratitis

Abstract

Repository corticotropin injection (RCI; Mallinckrodt Pharmaceuticals) is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides approved for the treatment of keratitis. This analysis of topline patient-reported outcome results from a multicenter, open-label, phase 4 study of RCI in patients with refractory severe noninfectious keratitis was conducted to assess quality of life (QoL) and symptomatic improvements (NCT04169061). Patients were ≥18 years old with severe keratitis despite previous treatment with topical cyclosporine, lifitegrast, or an immunosuppressant. Patients received 80 U RCI subcutaneously twice weekly for 12 weeks. Analyses evaluated the change from baseline through week 12 on the Impact of Dry Eye on Everyday Life (IDEEL) questionnaire, Visual Analogue Scale (VAS), and Ocular Discomfort Score (ODS). One-sample t tests (null hypothesis: no change) were used for statistical analyses, with significance vs baseline determined as p<0.05. Analyses were performed using the modified intent-to-treat (mITT) population (patients who received ≥1 dose of RCI and contributed any postbaseline efficacy data). Topline results in the mITT population (N=35) showed significant improvements from baseline at every time point assessed after initiation of RCI treatment in 4 of 6 dimensions of the IDEEL questionnaire (ie, emotional impact, impact on daily activities, symptom bother, and treatment-related bother scores); significant improvements from baseline at weeks 6 and 12 were observed for the IDEEL impact on work score. VAS scores for eye discomfort, eye dryness, foreign body sensation, pain, and photophobia significantly improved at all measured time points after initiation of RCI. ODS showed significant improvements from baseline at every time point assessed after RCI initiation for the overall score and for the symptoms of dryness, grittiness, and stinging. Topline results of this open-label study showed that RCI significantly improved QoL and symptom measures in patients with refractory severe noninfectious keratitis.