A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I
研究概览
简要总结
This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference on Harmonization (ICH) guidelines. Eligible subjects satisfying all inclusion and none of the exclusion criteria will be enrolled. All subject who completed the parent clinical study (NSCT/CT/18/01) will undergo safety and efficacy assessments up to 5 years post study drug injection
详细描述
The observational study population comprises of individuals who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection in a parent study.
Enrolled subjects will undergo safety and efficacy assessments for up to 5 years from the vMCO-I administration. At the visits, subjects will have safety and efficacy evaluations. Subjects will have a final visit at 60 months post study drug administration.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The subject population includes subjects who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I), administered via intravitreal injection in a parent study.
排除标准
- •Subjects who will not consent for study.
结局指标
主要结局
Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I
时间窗: 20 Months
Assessments of Incidences, nature, and severity of treatment emergent adverse events (TEAEs), Serious Adverse Events (SAEs); intraocular inflammation graded through ocular exam; intraocular pressure and retinal anatomy
Evaluation of the long-term efficacy of a single intravitreal injection of Multi-Characteristic Opsin (vMCO-I)
时间窗: 20 Months
Assessment of treatment effect with the change from baseline in Freiburg Visual Acuity (quantitative LogMAR score)
次要结局
- Assessment of the effect of vMCO-I on functional vision outcomes(20 Months)
- Assessment of the durability of vMCO-I induced gene reporter expression(20 Months)