Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- The Best Corrected Visual Acuity(BCVA)
研究概览
简要总结
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
详细描述
Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited ocular disorder associated with a mutation in mtDNA . The common manifestation is visual loss which caused by the respiratory chain enzymes complex dysfunction resulting in increased oxidative stress enzymes production.
Material and Method Seven patients with 11778 LHON mutation were randomly treated with a Single IVT Injection of recombinant Adeno-Associated Virus-NADH dehydrogenase, subunit 4 (complex I)（rAAV2-ND4)（0.05ml).The dose was 5 × 10^9 vg/0.05 mL for patients younger than 12 years old, and 1 × 10^10 vg/0.05 mL for patients older than 12 years old. The visual acuity, visual evoked potential （VEP）,optical coherence tomography（ OCT）, computerized visual field, electroretinograms(ERG), retinal nerve fiber layer(RNFL)and Liver and kidney function in plasma were compared before and after treatment at 1,3,and 6, months interval.
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
- 8 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •comply with Leber hereditary optic neuropathy diagnostic criteria.
- •in patients with informed consent, voluntary participation.
- •signed informed consent.
- •8 ≤ Age ≤ 60 years old, good health, the patient can tolerate local anesthesia surgery.
- •to comply with doctor's instructions, can in the time of referral.
排除标准
- •Cardiopulmonary and renal function in severe weakness, cancer, a variety of bleeding disorders, acute sensing disease, high fever, high fever disease, women during pregnancy, heart disease, such as post-operative recovery period.
- •Are participating in other clinical studies of patients.
- •Patients with mental disorders.
研究组 & 干预措施
rAAV2-ND4
injection
干预措施: rAAV2-ND4 (Drug)
结局指标
主要结局
The Best Corrected Visual Acuity(BCVA)
时间窗: Up to 3 years
Results of CD3/CD4/CD8 Test
时间窗: up to 6 months
The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
次要结局
- Intraocular Pressure;(Up to 3 years)
- Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test(Up to 3 years)
- Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)(up to 3 years)
- Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)(up to 3 years)
- Neutralizing Antibody Assay(up to 3 years)
研究者
Bin Li
Safety and Efficacy Study of rAAV2-ND4 Treatment of LHON
Huazhong University of Science and Technology