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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

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 willing to sign a consent form 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 willing to sign a consent form 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 ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DRUG

Phase 1a: OCT-980 Capsule

OCT-980 capsules administered orally once at dose levels specified in the protocol

DRUG

Phase 1a: Placebo Capsule

Placebo capsules administered orally once at dose levels specified in the protocol

DRUG

Phase 1b/2: OCT-980 Tablet

OCT-980 tablets administered orally once at dose levels specified in the protocol

Locations (1)

CMAX Clinical Research Pty Ltd
Adelaide, South Australia, Australia