Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 14
- 试验地点
- 5
- 主要终点
- Incidence and severity of adverse events
研究概览
简要总结
This is an open-label, multiple ascending dose study to evaluate the safety, tolerability, and efficacy of EA-2353 in subjects with RP. Unilateral intravitreal injections (IVT) will be administered into the subject's Study Eye. There will be up to 4 cohorts.
详细描述
This is an open-label, multiple ascending dose study to evaluate the safety, tolerability, and efficacy of EA-2353 in subjects with RP. There will be up to 4 cohorts. Each of the first three cohorts will treat 3 subjects with 4 weekly injections of EA-2353.
Unilateral intravitreal injections (IVT) will be administered into the subject's Study Eye, which is determined as the eye with worse vision based on the BCVA. Eligible subjects will be enrolled into one of the following three cohorts in an ascending sequential fashion:
- Cohort 1 (low dose)
- Cohort 2 (mid dose)
- Cohort 3 (high dose)
- Cohort 4 (maximum tolerated dose)
Patient participation will last for approximately 25 months.
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
- •Male or female, ≥ 18 years of age.
- •Have a clinical and molecular diagnosis of RP.
- •Understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures.
- •Are willing to comply with the protocol and follow the instructions including attendance at all scheduled study visits.
- •BCVA in the worse eye between 20/50 and able to count fingers (CF)
- •Have clear ocular media.
- •Have pupillary dilation sufficient to allow for quality images.
排除标准
- •Subjects who are pregnant or suspected to be pregnant and subjects who are lactating or intend to breastfeed. Female subjects of childbearing potential are required to have a negative urine pregnancy test at screening and prior to each injection, and must agree to use an acceptable method of contraception from the time of signing informed consent until the end of the study.
- •Have any uncontrolled systemic disease or non-ocular disorder which would put the subject at risk due to study treatment or procedures, influence the results of the study, or impact the subject's ability to participate in the study (e.g., infection, uncontrolled elevated blood pressure, cardiovascular disease, and inability to maintain glycemic control).
- •Presence of a significant ocular disease or disorder in the Study Eye.
- •Have a history of any vitreoretinal surgery ever in the Study Eye.
- •Have received any prior cell or gene therapy for RP.
- •Have history or current abuse of alcohol and/or drugs.
研究组 & 干预措施
EA-2353
EA-2353 Ophthalmic Suspension will be administered via unilateral intravitreal injection into the Study Eye weekly for one month (4 injections)
干预措施: EA-2353 (Drug)
结局指标
主要结局
Incidence and severity of adverse events
时间窗: through study completion at 24 months
Incidence and severity of dose limiting toxicities (DLTs)
时间窗: through study completion at 24 months
Establish the maximum tolerated dose (MTD) as determined by occurrence of DLTs
时间窗: through study completion at 24 months
次要结局
未报告次要终点