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

NCT07408232 | Active Not Recruiting Phase 1

• 1 other identifier: OCT-980-101
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Retinitis Pigmentosa

Keywords

Rhodopsin-associated autosomal dominant Retinitis Pigmentosa; RHO-adRP; Retinitis Pigmentosa; Inherited Retinal Disease; autosomal dominant Retinitis Pigmentosa; Retinitis Pigmentosa 4; RP4; RP; Eye Diseases

Outcome Measures

Primary Outcomes (1)

• 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)

Study Arms (3)

Phase 1a: OCT-980

EXPERIMENTAL

OCT-980 capsules administered orally once daily under fasted or fed conditions per protocol

Drug: Phase 1a: OCT-980 Capsule

Phase 1a: Placebo

PLACEBO COMPARATOR

Matching placebo capsules administered orally once daily under fasted or fed conditions per protocol

Drug: Phase 1a: Placebo Capsule

Phase 1b/2: OCT-980

EXPERIMENTAL

OCT-980 tablets administered orally once daily.

Drug: Phase 1b/2: OCT-980 Tablet

Interventions

Phase 1a: OCT-980 Capsule DRUG

OCT-980 capsules administered orally once at dose levels specified in the protocol

Phase 1a: OCT-980

Phase 1a: Placebo Capsule DRUG

Placebo capsules administered orally once at dose levels specified in the protocol

Phase 1a: Placebo

Phase 1b/2: OCT-980 Tablet DRUG

OCT-980 tablets administered orally once at dose levels specified in the protocol

Phase 1b/2: OCT-980

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Octant, Inc.: lead

Study Sites (1)

CMAX Clinical Research Pty Ltd

Adelaide, South Australia, 5000, Australia

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Retinitis Pigmentosa 4; Eye Diseases

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Phase 1a: Double (Participant, Investigator); Phase 1b/2: Open Label
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2026
• First Posted: February 13, 2026
• Study Start: March 10, 2026
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): November 1, 2028
• Last Updated: May 19, 2026

Record last verified: 2026-05

Locations

AU (1)