Overview
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.
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 in healthy volunteers, which was conducted in Australia and is now complete.
The phase 1b/2 study is an 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).
RHO-adRP participants are currently being recruited for the phase 1b/2 study in the US.
Eligibility
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
