Retinitis Pigmentosa
Conditions
Keywords
Rhodopsin-associated autosomal dominant Retinitis Pigmentosa, RHO-adRP, Retinitis Pigmentosa, Inherited Retinal Disease, autosomal dominant Retinitis Pigmentosa, Retinitis Pigmentosa 4, RP4, RP, Eye Diseases
Brief summary
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.
Detailed 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.
Interventions
DRUGPhase 1b/2: Cohort 1 - OCT-980 Tablet
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
DRUGPhase 1b/2: Cohort 2 - OCT-980 Tablet
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
DRUGPhase 1b/2: Cohort 3 - OCT-980 Tablet
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
DRUGPhase 1b/2: Cohort 4 - OCT-980 Tablet
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
DRUGPhase 1b/2: Cohort 5 - OCT-980 Tablet
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
Sponsors
Octant, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Phase 1b/2 in RHO-adRP participants is being conducted in the US.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability of multiple doses (Phase 1b/2) of OCT-980 in participants with a genetic diagnosis of RHO-adRP. | Time Frame: From Baseline to Week 60 (Ph 1b/2) |
Countries
United States
Contacts
CONTACTOctant adRP Clinical Operations
Outcome results
None listed