OCT-980 oral treatment for rhodopsin-associated autosomal dominant retinitis pigmentosa

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

Phase 1b/2 is active. Inclusion

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 greater than or equal to age 18 (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) \> 55
* 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

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 it's running

• Casey Eye Institute
  Portland, Oregon, United States Recruiting
• Retina Foundation of the Southwest
  Dallas, Texas, United States Recruiting

Study contacts

• Study coordinator: Octant adRP Clinical Operations
• Email: adrpclinicaltrials@octant.bio
• Phone: +1-510-500-5956

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.