NCT02064569已完成1 期
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Incidence of local and general adverse events and Serious Adverse Events
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented diagnosis of LHON based on a genetic test confirming the presence of the G11778A mutation in the mitochondrial ND4
- •Age 18 years old or older at the time of study entry (informed consent signature)
- •Visual acuity ≤ 1/10 of the less functional eye
排除标准
- •Any known allergy or hypersensibility to one of the product used during the trial
- •Contraindication to IVT surgery (anaemia Hb <8g/dl, severe cardiovascular disease, severe coagulopathy…)
- •Disorder of the ocular humors and of the internal retina involving visual disability
- •Presence of other pathology whose symptoms or associated treatments might affect the retina or the optic nerve Vascular retinal occlusion
- •Narrow angle contra-indicating pupillary dilation
- •Other cause of optic neuropathy (inflammatory conditions or exposure to toxins...)
- •Patients presenting known mutation of other genes implicated in pathological retinal conditions
研究组 & 干预措施
GS010
Experimental
干预措施: GS010 (Genetic)
结局指标
主要结局
Incidence of local and general adverse events and Serious Adverse Events
时间窗: Up to 48 weeks
次要结局
未报告次要终点
研究者
研究点 (1)
Loading locations...
相似试验
已完成
1 期
Safety,Tolerability and MTD KA2507 (HDAC6 Inhibitor) in Patients With Solid TumoursSolid Tumor, AdultNCT03008018Karus Therapeutics Limited20
已完成
1 期
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)Spinal Muscular AtrophyNCT01494701Biogen28
已完成
1 期
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult VolunteersHealthyNCT04601844Regeneron Pharmaceuticals19
终止
1 期
Phase I Study of TENPA in Advanced Solid CancerAdvanced Solid TumorNCT02979392Yung-Jue Bang19
已完成
1 期
Study to Evaluate Safety, Efficacy of Intralesional Injection of STP705 in Adults With Facial isSCCFacial isSCCNCT05421013Sirnaomics9