Phase 1 Study to Check Safety of Single Dose ER-100 in Adults with Open Angle Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy

What this Trial Is About

Researchers are evaluating the safety and tolerability of ER-100, an investigational epigenetic therapy, in adults with optic nerve conditions including Open Angle Glaucoma OAG and Non-Arteritic Anterior Ischemic Optic Neuropathy NAION. This first-in-human Phase 1 trial aims to understand if a single dose of ER-100 is safe and what side effects may occur. ER-100 targets cellular aging in retinal cells by delivering genetic instructions for proteins that may help restore cell function without altering existing genes. Participants with OAG will be enrolled in a dose escalation phase testing low and high doses of ER-100 administered to one eye via a modified adeno-associated virus vector. Systemic doxycycline is given for 8 weeks to activate the therapy. After safety data review, a selected dose will be given to participants with NAION in a dose expansion phase. The study involves a sentinel participant at each dose level, monitored for 28 days before more participants are treated. Up to 18 participants are planned, with 15 clinic visits over 5 years and some phone visits. During the study, participants will have detailed eye exams, laboratory tests, and body fluid samples collected to monitor how ER-100 is processed and its effects. Questionnaires will assess quality of life. Safety will be closely monitored through various eye function tests and blood work during the first 112 days, then followed annually for up to 5 years. The main outcomes include tracking adverse events, changes in vision, eye pressure, and laboratory safety measures compared to baseline assessments before treatment.

Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions)