A Phase 1/2 Study to Assess Safety, Pharmacokinetics, and Effects of OCT-980 in Healthy Volunteers and People With Autosomal Dominant Retinitis Pigmentosa

What this Trial Is About

Researchers are evaluating OCT-980, an oral small molecule treatment, for patients with rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), a progressive eye disease causing vision loss. This Phase 1/2 study aims to assess the safety, tolerability, how the body absorbs and responds to the drug, and its effectiveness in both healthy volunteers and patients diagnosed genetically with RHO-adRP. There is currently no approved cure or treatment for this condition. The study includes two parts: Phase 1a conducted in Australia involves a single-dose, double-masked, placebo-controlled dose escalation to evaluate safety and drug behavior in healthy volunteers. Phase 1b/2 in the US involves multiple ascending doses of OCT-980 taken orally over up to 48 weeks in participants with RHO-adRP. The Phase 1b/2 part is open-label and multi-center, focusing on longer-term safety, tolerability, drug effects, and efficacy. Participants will undergo various assessments, including blood sampling for drug levels and safety labs, vision testing, and health evaluations. The primary outcome measures include safety and tolerability from baseline to day 5 for Phase 1a and up to week 60 for Phase 1b/2. The study monitors how participants respond to the medication over time with follow-up visits and laboratory tests to ensure safety and to evaluate treatment effects.

A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)