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, 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).
Eligibility
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
