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 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.
⁃ 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