Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 34
- 试验地点
- 2
- 主要终点
- Incidence of Adverse Events Related to the Treatment
研究概览
简要总结
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.
详细描述
The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 3 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Low dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single low dose of AAV-CNGA3 or AAV-CNGB3
干预措施: Prior exposure to AAV-CNGA3 or AAV-CNGB3 (Biological)
Intermediate dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single intermediate dose of AAV-CNGA3 or AAV-CNGB3
干预措施: Prior exposure to AAV-CNGA3 or AAV-CNGB3 (Biological)
Other dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single other dose (between the intermediate and high dose) of AAV-CNGA3 or AAV-CNGB3
干预措施: Prior exposure to AAV-CNGA3 or AAV-CNGB3 (Biological)
High dose of AAV-CNGA3 or AAV-CNGB3
Subretinal administration of a single high dose of AAV-CNGA3 or AAV-CNGB3
干预措施: Prior exposure to AAV-CNGA3 or AAV-CNGB3 (Biological)
结局指标
主要结局
Incidence of Adverse Events Related to the Treatment
时间窗: 5 Years
The primary outcome measure is the longer-term safety of treatment with AAV-CNGA3 or AAV-CNGB3, assessed by the absence of IMP-related adverse events.
次要结局
- Improvements in Visual Function as Assessed by Visual Acuity at Month 12(12 months)
- Improvements in Visual Function as Assessed by Visual Acuity at Month 60(60 months)
- Improvements in Retinal Function as Assessed by Static Perimetry at Month 12(12 months)
- Improvements in Retinal Function as Assessed by Static Perimetry at Month 60(60 months)
- Quality of Life at Month 12 Measured by QoL Questionnaires in Children and Adolescents(12 months)
- Quality of Life at Month 60 Measured by QoL Questionnaires in Children and Adolescents(60 months)
- Quality of Life at Month 12 Measured by QoL Questionnaires in Adults(12 months)
- Quality of Life at Month 60 Measured by QoL Questionnaires in Adults(60 months)