Phase 1 Study of C.001 in Retinal Degeneration
A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)
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.
研究概览
详细说明
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.
研究类型
注册 (估计的)
阶段
- 阶段1
联系人和位置
学习联系方式
- 姓名：Clinical Trial Administrator
- 电话号码：999-999-9999
- 邮箱：trials@celliotherapeutics.com
学习地点
-
-
California
-
Beverly Hills、California、美国、90210
- 招聘中
- Retina Vision Consultants
-
接触：
- Pradeep Prasad, MD
- 电话号码：(310) 269-8565
- 邮箱：trials@celliotherapeutics.com
-
Palo Alto、California、美国、94301
- 尚未招聘
- Palo Alto Medical Foundation
-
接触：
- Ehsan Rahimy, MD
- 电话号码：(650) 853-2974
- 邮箱：trials@celliotherapeutics.com
-
Sacramento、California、美国、95825
- 尚未招聘
- Retinal Consultants Medical Group
-
接触：
- David Telander, MD
- 电话号码：(916) 454-4861
- 邮箱：trials@celliotherapeutics.com
-
-
参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
描述
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：非随机化
- 介入模型：顺序分配
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Arm 1
C.001Low Dose
|
C.001 administered once via subretinal injection
|
|
实验性的：Arm 2
C.001 Medium Dose
|
C.001 administered once via subretinal injection
|
|
实验性的：Arm 3
C.001 High Dose
|
C.001 administered once via subretinal injection
|
研究衡量的是什么？
主要结果指标
结果测量 |
大体时间 |
|---|---|
|
Incidence of Ocular and Systemic Adverse Events
大体时间：3 months
|
3 months
|
次要结果测量
结果测量 |
大体时间 |
|---|---|
|
Change in visual function measured in EDTRS letters
大体时间：3 months
|
3 months
|
|
Findings of anatomic change on optical coherence tomography (OCT)
大体时间：3 months
|
3 months
|
合作者和调查者
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他研究编号
- CT-C.001-001
- CLIN2-19416 (其他赠款/资助编号：California Institute of Regenerative Medicine)
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
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