A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 54
- 试验地点
- 8
- 主要终点
- Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit
研究概览
简要总结
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.
详细描述
Phase 1 of the study includes up to 4 planned dose levels to be administered across up to 4 cohorts. Participants are assigned to receive OpCT-001 in an open-label manner.
Dose escalation in Phase 1 is being conducted using a standard 3+3 scheme in which a total of up to 24 legally blind participants (~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either dose-level cohort. Phase 2 participants and investigators and study site personnel outside of the surgical team will be masked to the Phase 2 OpCT-001 dose-level assignments.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
盲法说明
Phase 1: None (Open Label) Phase 2: Investigators and study site personnel outside of the surgical team will be masked to dose level assignments
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed genetic diagnosis of primary photoreceptor (PR) disease
- •Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.
- •BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.
- •Retinal structure examination in the study eye demonstrating regions suitable for cell administration.
排除标准
- •Clinically relevant, active ocular inflammation or infection
- •Glaucoma or other significant optic neuropathy
- •Diabetic macular edema or diabetic retinopathy
- •Clinically significant cystoid macular edema
- •In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia
- •Ocular surgery ≤3 months before Screening
- •Monocular vision (ie, no light perception in the fellow eye)
- •Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.
- •Any current and active infection (bacterial/viral/fungal) that could put the participant at risk from immunosuppression
- •History of any cell therapy, gene therapy, or retinal implant at any time
- •Previously received a bone marrow or solid organ transplant
研究组 & 干预措施
Cohort 1
OpCT-001 dose level 1 will be administered via subretinal injection.
干预措施: OpCT-001 (Biological)
Cohort 3 (optional)
OpCT-001 dose level 3 will be administered via subretinal injection.
干预措施: OpCT-001 (Biological)
Cohort 2
OpCT-001 dose level 2 will be administered via subretinal injection.
干预措施: OpCT-001 (Biological)
Cohort 4
OpCT-001 dose level 4 will be administered via subretinal injection.
干预措施: OpCT-001 (Biological)
结局指标
主要结局
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit
时间窗: From OpCT-001 administration through the Week 52 visit
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit
时间窗: From OpCT administration through the Week 52 visit
次要结局
- Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT)(From baseline through the Week 52 visit)
- Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT)(From enrollment through the Week 52 visit)