A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Retinoschisin (rAAV2tYF-CB-hRS1) in Patients With X-linked Retinoschisis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 27
- 试验地点
- 18
- 主要终点
- Number of Participants Experiencing Adverse Events
研究概览
简要总结
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis. Up to 27 participants will be enrolled and 3 dose levels will be evaluated in a dose escalation format.
详细描述
This will be a non-randomized, open label, Phase 1/2 dose escalation study.
Up to 27 participants will be enrolled. Each participant will receive the study agent by intravitreal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). Participants in the dose escalation phase will be ≥ 18 years of age. After the maximum tolerated dose is identified individuals ≥ 6 years of age will be enrolled.
Safety will be measured by the number and proportion of participants experiencing adverse events and immune response to RS1. Efficacy will be measured by evaluation of changes in visual function and schisis cavity size.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 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
- Male
- 接受健康志愿者
- 否
入选标准
- •Retinal disease consistent with a diagnosis of XLRS and documented mutations in the RS1 gene
- •Male individual at least 18 years of age (dose escalation phase) or at least 6 years of age (maximum tolerated dose phase),
- •Able to perform tests of visual and retinal function,
- •Visual acuity specified for each group
- •Not treated with CAIs currently or within 3 months prior to study enrollment,
- •Have acceptable laboratory parameters.
排除标准
- •Prior receipt of any AAV gene therapy product,
- •Pre-existing eye conditions that would preclude the planned intravitreal injection or interfere with interpretation of study endpoints or complications of vector administration.
结局指标
主要结局
Number of Participants Experiencing Adverse Events
时间窗: From Day 0 to Month 12 (12 months)
Number of participants with ocular adverse events, assessed by standard ophthalmic examination, including slit lamp biomicroscopy, tonometry, and indirect ophthalmoscopy.
次要结局
- Change From Baseline in Schisis Cavity Size on Optical Coherence Tomography (OCT)(From Day 0 to Month 12 (12 months))
- Change From Baseline in B-wave Amplitude in Electroretinogram (ERG) Responses(From Day 0 to Month 12 (12 months))
- Change From Baseline in Best Corrected Visual Acuity (BCVA)(From Day 0 to Month 12 (12 Months))