NCT06196827进行中（未招募）1 期
A Multi-center Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of rAAV2-RPE65 Gene Therapy (LX101) in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy
Innostellar Biotherapeutics Co.,Ltd2 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2022年7月2日最近更新:
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Incidence of dose-limiting toxicity (DLT)
研究概览
简要总结
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject and/or their guardian signing a written informed consent.
- •Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- •Subjects are 6 years of age or older.
- •Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.
排除标准
- •Prior gene therapy for IRD and other hereditary eye diseases.
- •Pre-existing eye conditions that would interfere with interpretation of study endpoints.
- •Active intraocular or periocular infections in the study eye.
- •Lacking of sufficient surviving retinal cells.
- •Prior ocular surgery within six months.
- •Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
- •Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds.
研究组 & 干预措施
LX101 Dose 1
Experimental
干预措施: LX101 (Genetic)
LX101 Dose 2
Experimental
干预措施: LX101 (Genetic)
结局指标
主要结局
Incidence of dose-limiting toxicity (DLT)
时间窗: 1 month
Incidence of DLT following LX101 subretinal injection at different doses
Incidence of adverse events (AEs) and serious adverse events (SAEs)
时间窗: 12 months
Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection
次要结局
- Efficacy of LX101 in study eye(12 months)
研究者
研究点 (2)
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