An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 23
- 试验地点
- 3
- 主要终点
- Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.
研究概览
简要总结
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
详细描述
CNGB3 retinal gene therapy for patients with achromatopsia
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Are aged 3 years or older
- •Have achromatopsia confirmed by a retinal specialist (CI or PI)
排除标准
- •Are females who are pregnant or breastfeeding
- •Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
- •Have any other condition that the CI/PI considers makes them inappropriate for entry into the trial
结局指标
主要结局
Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.
时间窗: 6 weeks
The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences * Severe unresponsive inflammation * Infective endophthalmitis * Ocular malignancy * Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
次要结局
- Improvements in Visual Function as Assessed by Visual Acuity(6 months)
- Improvements in Retinal Function as Assessed by Static Perimetry(6 months)
- Quality of Life Measured by QoL Questionnaires in Children and Adolescents(6 months)
- Quality of Life Measured by QoL Questionnaires in Adults(6 Months)