NCT01208389进行中（未招募）1 期
A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Adverse events as a measure of safety and tolerability
研究概览
简要总结
The study is a follow-on to a Phase 1 dose-escalation and safety study.
详细描述
The study is a follow-on to a Phase 1 dose-escalation and safety study (closed to enrollment as of June 2009). Up to twelve adults and children with a molecular diagnosis of biallelic RPE65 mutations, who have participated in the earlier Phase 1 study, and who meet all study eligibility criteria, will receive AAV2-hRPE65v2 vector in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of AAV2-hRPE65v2.
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 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prior participation in Phase 1 study with unilateral, subretinal administration of AAV2-hRPE65v
- •Visual acuity equal to or greater than light perception.
- •Sufficient viable retinal cells in contralateral, previously uninjected eye, as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy. Must have either: 1) an area of retina within the posterior pole of > 100 µm shown on OCT; 2) ≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole; or 3) remaining visual field within 50 degrees of fixation.
- •Willingness to adhere to protocol and long-term follow-up as evidenced by written informed consent or parental permission and subject assent (where applicable).
排除标准
- •Unable or unwilling to meet requirements of the study.
- •Participation in any other study of an investigational drug within the past six months.
- •Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme; individuals who discontinue use of these compounds for 18 months may become eligible.
- •Prior intraocular surgery within six months.
- •Known sensitivity to medications planned for use in the peri-operative period.
- •Pre-existing eye conditions, such as glaucoma, or complicating systemic diseases that would preclude the planned surgery or could interfere with the interpretation of study. Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example: radiation treatment of the orbit; leukemia with CNS/optic nerve involvement). Subjects with diabetes or sickle cell disease would be excluded if they had any manifestation of advanced retinopathy (e.g. macular edema or proliferative changes). Also excluded would be subjects with immunodeficiency (acquired or congenital) as there could be susceptibility to opportunistic infection (such as CMV retinitis).
- •Individuals of childbearing potential who are pregnant or unwilling to use effective contraception for four months following vector administration.
- •Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.
- •Subjects will NOT be excluded based on their gender, race, or ethnicity.
研究组 & 干预措施
voretigene neparvovec-rzyl (AAV2-hRPE65v2)
Experimental
Administration of study agent (AAV2-hRPE65v2) to the previously, uninjected contralateral eye:
干预措施: voretigene neparvovec-rzyl (Biological)
结局指标
主要结局
Adverse events as a measure of safety and tolerability
时间窗: 15 years
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
次要结局
- Visual acuity(15 years)
- Pupillary light response(15 years)
- Full-field light threshold sensitivity testing(15 years)
- Visual field(15 years)
- Mobility testing(15 years)
- Contrast sensitivity(15 years)
研究者
研究点 (1)
Loading locations...
相似试验
终止
1 期
AZD7325 Japan Multiple Ascending Dose (MAD) StudyHealthyNCT00945425AstraZeneca48
已完成
1 期
Follow-up Study of Safety and Efficacy in Subjects Who Completed NEUROSTEM® Phase-I/IIa Clinical Trial.Alzheimer's DiseaseNCT03172117Medipost Co Ltd.36
进行中（未招募）
1 期
Study of AR-14034 in Participants With Neovascular Age-Related Macular Degeneration (nAMD)Neovascular Age-related Macular Degeneration (nAMD)NCT05769153Alcon Research139
已完成
1 期
A Study of XmAb®22841 Monotherapy & in Combination w/ Pembrolizumab in Subjects w/ Selected Advanced Solid TumorsCervical CarcinomaPancreatic CarcinomaTriple Negative Breast CancerUrothelial CarcinomaSquamous Cell Carcinoma of the Head and NeckHepatocellular CarcinomaMelanomaRenal Cell CarcinomaNon-small Cell Lung CarcinomaSmall Cell Lung CarcinomaGastric or Gastroesophageal Junction AdenocarcinomaAdvanced or Metastatic Solid TumorsProstate CarcinomaFallopian Tube CancerPrimary Peritoneal CarcinomaSquamous Cell Anal CancerSquamous Cell Penile CarcinomaSquamous Cell Vulvar CarcinomaColorectal CarcinomaEndometrial CarcinomaIntrahepatic CholangiocarcinomaEpithelial Ovarian CancerNasopharyngeal CarcinomaNCT03849469Xencor, Inc.78
已完成
1 期
AZD2624 Multiple Ascending Dose Study in JapanHealthySafetyNCT00696865AstraZeneca30