NCT07408232

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

A Phase 1/2, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of OCT-980 in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Octant, Inc. · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Accepted

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, to be conducted in Australia; and a Phase 1b/2, to be conducted in the US. The phase 1a study will be a single dose, double-masked, placebo-controlled ascending dose escalation (SAD +/- food effect) to evaluate the safety, tolerability, and PK of OCT-980 in healthy volunteers. The phase 1b/2 study will be 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).

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
DRUG	Phase 1a: OCT-980 Capsule	OCT-980 capsules administered orally once at dose levels specified in the protocol
DRUG	Phase 1a: Placebo Capsule	Placebo capsules administered orally once at dose levels specified in the protocol
DRUG	Phase 1b/2: OCT-980 Tablet	OCT-980 tablets administered orally once at dose levels specified in the protocol

Timeline

• Start date: 2026-02-09
• Primary completion: 2028-11-01
• Completion: 2028-11-01
• First posted: 2026-02-13
• Last updated: 2026-02-13

Locations

1 site across 1 country: Australia