A Phase1/2a, Open-Label Study to Evaluate the Safety and Tolerability of RPE Stem Cell-derived RPE (RPESC-RPE) Transplantation as Therapy for Dry Age-related Macular Degeneration (AMD)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 3
- 主要终点
- Safety and tolerability of RPESC-RPE-4W transplantation
研究概览
简要总结
The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.
详细描述
RPESC-RPE-4W is Allogeneic RPE stem cell (RPESC)-derived RPE cells (RPESC-RPE) isolated from the RPE layer of human cadaveric eyes are transplanted under the macular.
This first-in-human Phase 1/2a open-label dose-escalation interventional study plans to enroll a total of 18 subjects
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
入排标准
- 年龄范围
- 55 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of dry AMD.
- •Ability to understand and give informed consent.
- •Adult male or female >55 years of age.
- •Medically suitable to undergo vitrectomy and subretinal injection (>60% on Karnofsky scale).
- •Postmenopausal if female (expected to be common for the age limitation), or the female partner of a male subject unable to father children.
- •If male, willing to use barrier and spermicidal contraception during the study.
排除标准
- •Allergy or hypersensitivity to dilation drops or fluorescein.
- •Active major medical conditions limiting ability to participate in the study.
- •Active malignancy or treatment with chemotherapy.
- •Systemic immunosuppressant therapy within past six months.
- •History of toxoplasmosis, retinal histoplasmosis or tuberculosis.
- •Receipt of investigational product (IP) in a clinical trial within prior six months.
- •Any other medical condition, which, in the Investigator's judgment, will interfere with the subject's ability to comply with the protocol, compromises subject safety, or interferes with the interpretation of the study results.
- •Pregnant or nursing females.
研究组 & 干预措施
50,000 cells
Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.
干预措施: RPESC-RPE-4W (Biological)
150,000 cells
Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.
干预措施: RPESC-RPE-4W (Biological)
250,000 cells
Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.
干预措施: RPESC-RPE-4W (Biological)
结局指标
主要结局
Safety and tolerability of RPESC-RPE-4W transplantation
时间窗: 24 months
The transplantation of RPESC-RPE-4W cells will be considered safe and tolerated in the absence of: * Decrease in visual acuity (VA) of more than 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (or to worse than counting fingers at three feet) from baseline * Any Grade 2 (CTCAE v5.0) or greater non-ocular Adverse Events (AE) related to the RPESC-RPE-4W investigational product (IP) or investigational interventions * Intraocular inflammation indicating contamination with an infectious agent or serious immune response greater than moderate severity related to the RPESC-RPE-4W investigational product or investigational interventions * Tumor formation \> 1 mm size related to the IP or investigational interventions
次要结局
- Change in the mean of Best Corrected Visual Acuity (BCVA)(24 months)
- Change in GA lesion area(24 months)
- Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry(24 months)
- Evidence of structural changes(24 months)