NCT03140969已完成1 期
An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 3
- 主要终点
- Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
详细描述
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation. Subjects will receive QR-110 in one eye every 3 months, for a maximum of 4 doses. Up to 3 dose levels of QR-110 will be evaluated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 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
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, ≥ 6 years of age at Screening with a clinical diagnosis of LCA and a molecular diagnosis of homozygosity or compound heterozygosity for the CEP290 p.Cys998X mutation.
- •Best-corrected visual acuity greater than or equal to light perception in both eyes and equal to or worse than LogMAR +1.0 (Snellen notation 20/200) in the worse eye and equal to or worse than LogMAR +0.7 (Snellen notation 20/100) in the contralateral eye.
- •Detectable outer nuclear layer (ONL) in the area of the macula.
- •An electroretinogram (ERG) result consistent with LCA.
- •Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging.
排除标准
- •Syndromic disease.
- •Pregnant or breast-feeding female.
- •Any clinically significant cardiac disease or defect.
- •One or more coagulation parameters outside of the normal range.
- •Any ocular disease or condition that could compromise treatment safety, visual acuity or interfere with assessment of efficacy and safety.
- •Prior receipt of intraocular surgery or intravitreal injection within 3 months prior to study start or planned intraocular surgery or procedure during the course of the study.
- •Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the PQ-110-001 study period.
- •Any prior receipt of genetic therapy for LCA
研究组 & 干预措施
QR-110
Experimental
Administered every 3 months
干预措施: QR-110 (Drug)
结局指标
主要结局
Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes
时间窗: 1 year
次要结局
- Frequency and Severity of Non-ocular Adverse Events(1 year)
- Change in Best-corrected Visual Acuity (BCVA)(1 year)
- Change in Full-field Stimulus Test (FST)(1 year)
研究者
研究点 (3)
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