OCT-980 oral treatment for rhodopsin-associated autosomal dominant retinitis pigmentosa
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 program will test whether the oral drug OCT-980 is safe, well tolerated, and shows signs of benefit in adults with RHO-associated autosomal dominant retinitis pigmentosa and in healthy volunteers.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Octant, Inc. Industry-sponsored |
| Locations | 1 site (Adelaide, South Australia) |
| Trial ID | NCT07408232 on ClinicalTrials.gov |
What this trial studies
Octant is testing OCT-980, an oral small-molecule corrector designed to target misfolded rhodopsin in RHO-associated autosomal dominant retinitis pigmentosa (RHO-adRP). The program includes a Phase 1a single-ascending-dose, randomized, double-masked, placebo-controlled healthy volunteer trial in Australia with a food-effect cohort, followed by a Phase 1b/2 open-label, multiple-ascending-dose study in the U.S. that will enroll adults with genetically confirmed RHO-adRP for up to 48 weeks. Key outcomes include safety, tolerability, pharmacokinetics and pharmacodynamics, with exploratory efficacy measures in the patient cohort. The goal is to determine dose ranges and early signals of clinical activity for future development.
Who should consider this trial
Good fit: Ideal candidates are adults (18–65) with a genetically confirmed diagnosis of rhodopsin-associated autosomal dominant retinitis pigmentosa for the Phase 1b/2 portion, and healthy adults meeting protocol criteria for the Phase 1a portion.
Not a fit: Patients without RHO mutations, those with very advanced/near-total retinal degeneration, children, or people who do not meet age, weight, or other protocol eligibility criteria are unlikely to benefit from participation.
Why it matters
Potential benefit: If successful, OCT-980 could slow or stabilize vision loss in people with RHO-related autosomal dominant retinitis pigmentosa by correcting defective rhodopsin protein function.
How similar studies have performed: This is a relatively novel, mechanism-based approach: preclinical data and early-phase work support small-molecule rhodopsin correctors, but no approved therapies exist and clinical benefit remains unproven.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Adelaide, South Australia
- CMAX Clinical Research Pty Ltd — Adelaide, South Australia, Australia (Recruiting)
Study contacts
- Study coordinator: Octant adRP Clinical Operations
- Email: adrpclinicaltrials@octant.bio
- Phone: +1-510-500-5956
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.