NCT07054632进行中（未招募）3 期
Efficacy and Safety of Gene Therapy rAAV-RPE65 (LX101) in Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy: a Phase III, Multicenter, Randomized Controlled Trial (STAR)
Innostellar Biotherapeutics Co.,Ltd4 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2023年9月13日最近更新:
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 30
- 试验地点
- 4
- 主要终点
- Mobility Test
研究概览
简要总结
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- 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.
- •Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
研究组 & 干预措施
Intervention group
Experimental
干预措施: LX101 (Genetic)
Control group
No Intervention
结局指标
主要结局
Mobility Test
时间窗: 12 months
Changes in functional vision from baseline, determined by mobility test score
次要结局
- Mobility Test(6 months)
- Full-field Light Sensitivity Threshold (FST) Test(6 months、12 months)
- Visual Acuity(6 months、12 months)
- Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)(12 months)
研究者
研究点 (4)
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