NCT04516369已完成3 期
An Open-label, Single-arm Study to Provide Efficacy and Safety Data of Voretigene Neparvovec Administered as Subretinal Injection in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Change from Baseline in full-field light sensitivity threshold
研究概览
简要总结
The purpose of this study is to provide safety and efficacy data for voretigene neparvovec, administered as subretinal injection, in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy.
详细描述
This is an open-label, single-arm study to evaluate the safety and efficacy of bilateral subretinal administration of voretigene neparvovec in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include full-field light sensitivity threshold testing, visual fields, visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.
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
- 4 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Japanese participants with biallelic RPE65 mutation-associated retinal dystrophy; molecular diagnosis of RPE65 mutation must be confirmed by a Novartis designated laboratory in Japan.
- •Age four years or older.
- •Visual acuity worse than 20/60 (both eyes) and/or visual field less than 20 degrees in any meridian as measured by a III4e isopter or equivalent (both eyes).
- •Sufficient viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/ or ophthalmoscopy. Must have either:
- •An area of retina within the posterior pole of > 100 µm thickness shown on OCT, or
- •≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole, or
- •Remaining visual field within 30 degrees of fixation as measured by a III4e isopter or equivalent
排除标准
- •Any prior participation in a study in which a gene therapy vector was administered.
- •Participation in a clinical study with an investigational drug in the past 6 months from screening visit.
- •Known hypersensitivity to any of the study treatments including excipients or to medications planned for use in the peri-operative period.
- •Unable to reliably perform the FST assessment.
- •Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme in the past 6 months from screening visit.
- •Prior intraocular surgery within 6 months from screening visit.
- •Prior use of any medicines that, in the opinion of the investigator, may have caused retinal damage (e.g., sildenafil or related compounds, hydroxychloroquine, chloroquine, thioridazine, any other retino-toxic compounds)
- •Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery or 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.
研究组 & 干预措施
Voretigene neparvovec
Experimental
1.5 E11 vg (0.3 mL subretinal injection in each eye, 6-18 days apart)
干预措施: voretigene neparvovec (Genetic)
结局指标
主要结局
Change from Baseline in full-field light sensitivity threshold
时间窗: Baseline, Day 30, 90, 180, 270, and Year 1 after second eye injection
Full-field light sensitivity threshold (FST) is evaluated using white light, as averaged over both eyes.
次要结局
- Change from Baseline in macular threshold(Baseline, Day 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection)
- Change from Baseline in FST for long-term period(Baseline, Year 2, 3, 4 and 5 after second eye injection)
- Proportion of subject with the presence of vector shedding of voretigene neparvovec during the study period(Baseline, Day 0, 1 and 3 after first eye injection; Day 0, 1, 3, 14, 30, 90, 180, 270, and Year 1 after second eye injection)
- Change from Baseline in visual field(Baseline, Day 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection)
- Change from Baseline in visual acuity(Baseline, Day 1, and 3 after first eye injection; Day 1, 3, 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection)
- Proportion of subject with the presence of immunogenicity of voretigene neparvovec during the study period(Baseline, Day 30, 90, 180, 270, and Year 1 after second eye injection)
研究者
研究点 (2)
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