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
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