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
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- Safety and tolerability of single (Phase 1a) or multiple doses (Phase 1b/2) of OCT-980 in volunteers or participants with a genetic diagnosis of RHO-adRP, respectively
研究概览
简要总结
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.
详细描述
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, to be conducted in Australia; and a Phase 1b/2, to be conducted in the US.
The phase 1a study will be a single dose, double-masked, placebo-controlled ascending dose escalation (SAD +/- food effect) to evaluate the safety, tolerability, and PK of OCT-980 in healthy volunteers.
The phase 1b/2 study will be 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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
Phase 1a: Double (Participant, Investigator); Phase 1b/2: Open Label
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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)
排除标准
- •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
研究组 & 干预措施
Phase 1a: OCT-980
OCT-980 capsules administered orally once daily under fasted or fed conditions per protocol
干预措施: Phase 1a: OCT-980 Capsule (Drug)
Phase 1a: Placebo
Matching placebo capsules administered orally once daily under fasted or fed conditions per protocol
干预措施: Phase 1a: Placebo Capsule (Drug)
Phase 1b/2: OCT-980
OCT-980 tablets administered orally once daily.
干预措施: Phase 1b/2: OCT-980 Tablet (Drug)
结局指标
主要结局
Safety and tolerability of single (Phase 1a) or multiple doses (Phase 1b/2) of OCT-980 in volunteers or participants with a genetic diagnosis of RHO-adRP, respectively
时间窗: Time Frame: From Baseline to Day 5 (Phase 1a) or Week 60 (Ph 1b/2)
次要结局
未报告次要终点