A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 15
- 试验地点
- 6
研究概览
简要总结
This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium.
Participants will receive a single administration of C.001 delivered by subretinal injection. The study will evaluate three dose levels in a sequential manner.
The primary objective is to assess the safety of C.001 over a 3-month period following administration. Participants will be followed for up to 12 months to further evaluate safety and explore changes in visual function and retinal structure.
详细描述
This is a Phase 1, single-center, open-label, dose-escalation study designed to evaluate the safety and tolerability of C.001 administered via subretinal injection.
Participants will be enrolled into three sequential dose cohorts. A sentinel dosing strategy will be used, with safety review by an independent Data Safety Monitoring Board (DSMB) prior to escalation to higher dose levels. A minimum observation period of 4 weeks will be required between dose escalations.
Participants will be followed for up to 12 months after treatment for safety assessments and exploratory evaluation of visual and anatomical outcomes.
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
Open-Label
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
- •Age ≥18 years
- •Diagnosis of retinal disease associated with retinal pigment epithelium degeneration (e.g., geographic atrophy or Stargardt disease)
- •Visual acuity within protocol-defined limits
- •Stable ocular condition prior to enrollment
- •Medically suitable for ophthalmic surgery and anesthesia
- •Willingness to comply with study procedures and follow-up
排除标准
- •Active or prior choroidal neovascularization (CNV)
- •Recent myocardial infarction or significant uncontrolled cardiovascular disease
- •Uncontrolled glaucoma
- •Significant ocular inflammatory disease
- •Recent intraocular surgery within protocol-defined timeframe
- •Active systemic infection
- •Poorly controlled diabetes (e.g., HbA1c >8.5%)
- •Participation in another investigational study within 3 months prior to screening
研究组 & 干预措施
Arm 3
C.001 High Dose
干预措施: Injection (Biological)
Arm 1
C.001Low Dose
干预措施: Injection (Biological)
Arm 2
C.001 Medium Dose
干预措施: Injection (Biological)