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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Retinitis Pigmentosa
• Intervention / Treatment: Drug: Phase 1a: OCT-980 Capsule; Drug: Phase 1a: Placebo Capsule; Drug: Phase 1b/2: OCT-980 Tablet

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).

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Australia
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • CMAX Clinical Research Pty Ltd

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion

Phase 1a (Single Ascending Dose Escalation in Healthy Volunteers) Inclusion:

• Be in good general health, as determined by the Investigator
• Male or female age 18 to 65 years (inclusive) at the time of signing the informed consent form
• Have a body weight >50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m^2 (inclusive) at screening
• Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples
• Participants in Cohort 2b will be asked to participate in an additional food effect cohort and capable of consuming the high-fat meal within the protocol-specified time period and willing to consume 100% of the high-fat meal

Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Inclusion:

• Participants must have a diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa confirmed by a genetic test that is implemented in accordance with the relevant national requirements
• Male or female age 18 to 65 years (inclusive) at the time of signing the informed consent form
• Have a body weight >50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m^2 (inclusive) at screening
• Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) > 70
• Other than their diagnosis of RHO-adRP, be in good general health, as determined by the Investigator
• Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples Exclusion

Phase 1a (Single Ascending Dose Escalation in Healthy Volunteers) Exclusion:

• Females who are pregnant or breastfeeding
• Have evidence of any significant ocular or non-ocular disease/ disorder
• Has lifetime history of ocular surgery
• Has a history of major surgery, severe trauma, or bone fracture within 12 weeks before the first dose of the investigational product or planned surgery within 4 weeks after the end of the study

Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Exclusion:

• Females who are pregnant or breastfeeding
• Evidence of any other significant ocular (other than RHO-adRP) and non-ocular disease/ disorder
• Lifetime history of ocular surgery
• Any prior or current ophthalmologic gene therapy
• Has a history of major surgery, severe trauma, or bone fracture within 12 weeks before the first dose of the investigational product or planned surgery within 4 weeks after the end of the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 1a: OCT-980; OCT-980 capsules administered orally once daily under fasted or fed conditions per protocol	Drug: Phase 1a: OCT-980 Capsule; OCT-980 capsules administered orally once at dose levels specified in the protocol
Placebo Comparator: Phase 1a: Placebo; Matching placebo capsules administered orally once daily under fasted or fed conditions per protocol	Drug: Phase 1a: Placebo Capsule; Placebo capsules administered orally once at dose levels specified in the protocol
Experimental: Phase 1b/2: OCT-980; OCT-980 tablets administered orally once daily.	Drug: Phase 1b/2: OCT-980 Tablet; OCT-980 tablets administered orally once at dose levels specified in the protocol

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety and tolerability of single (Phase 1a) or multiple doses (Phase 1b/2) of OCT-980 in volunteers or participants with a genetic diagnosis of RHO-adRP, respectively	Time Frame: From Baseline to Day 5 (Phase 1a) or Week 60 (Ph 1b/2)

Collaborators and Investigators

• Sponsor: Octant, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 10, 2026
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): November 1, 2028

Study Registration Dates

• First Submitted: February 6, 2026
• First Submitted That Met QC Criteria: February 6, 2026
• First Posted (Actual): February 13, 2026

Study Record Updates

• Last Update Posted (Actual): May 19, 2026
• Last Update Submitted That Met QC Criteria: May 18, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Eye Diseases; RP; Retinitis Pigmentosa; Inherited Retinal Disease; Rhodopsin-associated autosomal dominant Retinitis Pigmentosa; RHO-adRP; autosomal dominant Retinitis Pigmentosa; Retinitis Pigmentosa 4; RP4
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases, Hereditary; Retinal Diseases; Retinal Dystrophies; Retinal Degeneration; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Eye Diseases; Retinitis Pigmentosa; Retinitis Pigmentosa 4
• Other Study ID Numbers: OCT-980-101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No