NCT00643747已完成1 期
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-onset Retinal Degeneration
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- intraocular inflammation
研究概览
简要总结
The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.
详细描述
The main objective of the proposed trial is to determine the safety and efficacy subretinal administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at three different dosage levels in individuals with autosomal recessive severe early-onset retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring plan to investigate the safety and efficacy of vector delivery.
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
- 5 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65
排除标准
- •Visual acuity in the study eye better than 6/36 Snellen
- •Hypertension
- •Diabetes mellitus
- •Tuberculosis
- •Renal impairment
- •Immunocompromise
- •Osteoporosis
- •Gastric ulceration
- •Severe affective disorder)
- •Pregnancy or lactation
结局指标
主要结局
intraocular inflammation
时间窗: at intervals up to 12 months
次要结局
- visual function(intervals up to 12 months)
研究者
研究点 (2)
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