A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Start Date
3/10/2026
Completion Date
11/1/2028
Summary
This integrated Ph1/2 clinical study is to assess the safety, tolerability and pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of OCT-980 in healthy volunteers and participants with RHO-adRP.
Detailed Description
Octant, Inc. is evaluating OCT-980, an oral small molecule corrector for the treatment of patients with rhodopsin (RHO)-associated autosomal dominant retinitis pigmentosa (RHO-adRP). There is no known cure and no currently approved therapy for RHO-adRP, a disease of the retina characterized by progressive loss of vision. This study is comprised of a Phase 1a in healthy volunteers, which was conducted in Australia and is now complete. The phase 1b/2 study is an open-label, multi-center multiple ascending dose escalation (MAD) to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of multiple-ascending doses of OCT-980 for up to 48 weeks, in participants with a genetic diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa (RP). RHO-adRP participants are currently being recruited for the phase 1b/2 study in the US.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
Phase 1b/2: Cohort 1 - OCT-980 Tablet
Phase 1b/2: Cohort 2 - OCT-980 Tablet
Phase 1b/2: Cohort 3 - OCT-980 Tablet
Phase 1b/2: Cohort 4 - OCT-980 Tablet
Phase 1b/2: Cohort 5 - OCT-980 Tablet
Conditions
Locations
Casey Eye Institute
Portland, Oregon 97239
United States
Retina Foundation of the Southwest
Dallas, Texas 75231-5080
United States