A Prospective, Multicenter, Open-Label, Single-Arm Study of the Safety and Tolerability of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- jCyte, Inc
- 入组人数
- 28
- 试验地点
- 3
- 主要终点
- Number of Subjects With Adverse Events as a Measure of Safety and Tolerability
研究概览
简要总结
This study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa. Four different dose levels of cells will be assessed in each of two groups of patients.
详细描述
The primary purpose of this study is to test the safety and tolerability of the administration of a single dose of jCell to adults with retinitis pigmentosa.
The goal of jCell therapy is to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated.
Human allogeneic retinal progenitor cells will be injected into adults with advanced RP to see if the procedure is safe, if the cells survive, and whether they have any impact on the visual status of the patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Clinical diagnosis of RP confirmed by electroretinogram (ERG) and willing to consent to mutation typing, if not already done
- •Best corrected visual acuity (BCVA) 20/63 or worse and no worse than hand motions (HM)
- •Adequate organ function and negative infectious disease screen
- •Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study
排除标准
- •Eye disease other than RP that impairs visual function
- •Pseudo-RP, cancer-associated retinopathies
- •History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents
- •Known allergy to penicillin or streptomycin
结局指标
主要结局
Number of Subjects With Adverse Events as a Measure of Safety and Tolerability
时间窗: 12 months
proportion of subjects with treatment emergent adverse events (TEAE), related TEAE and severe TEAE
次要结局
- Change in Mean Best Corrected Visual Acuity (BCVA)(12 months)