A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)

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.

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Key information

Conditions

Retinitis Pigmentosa Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Retinal Degeneration Retinal Diseases Retinal Dystrophies Retinitis Pigmentosa 4

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Casey Eye Institute, Portland, Oregon, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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