Clinical Research Directory

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.

Visual Impairment and Functional Visual Performance Following Acquired Brain Injury: A Cross-Sectional Study

This cross-sectional observational study aims to characterize acquired visual impairment in adults with acquired brain injury and to examine the relationship between objectively identified visual impairment and self-reported functional visual performance. A total of 108 adults with acquired brain injury, including stroke, traumatic brain injury, and brain tumor, will undergo a standardized visual assessment. Objective visual function will be evaluated across five predefined domains: visual acuity, contrast sensitivity, visual field, oculomotor function, and visual attention/scanning. Self-reported functional visual performance will be assessed using the Revised Self-Report Assessment of Functional Visual Performance (R-SRAFVP). The study will determine the proportion of participants with objectively identified visual impairment and examine the association between the R-SRAFVP score and the number of objectively impaired visual domains. The distribution of impairment across the individual visual domains will also be described.