NCT00747487已完成2 期
A Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Patients With Leber's Hereditary Optic Neuropathy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 85
- 试验地点
- 3
- 主要终点
- Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye
研究概览
简要总结
This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period.
详细描述
The study involves 6 clinic visits.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 14 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age > or = 14 years and < 65 years
- •Impaired visual acuity in at least one eye due to LHON
- •Onset of visual loss due to LHON lies five years or less prior to Baseline
- •Confirmation of either G11778A, T14484C or G3460A LHON mtDNA mutations at >60% in blood
- •No explanation for the visual failure besides LHON
- •Body weight ≥ 45 kg
- •Negative urine pregnancy test at Screening and at Baseline (women of childbearing potential).
排除标准
- •Treatment with Coenzyme Q10 or idebenone within 1 month prior to Baseline
- •Pregnancy and/or breast-feeding
- •Weekly alcohol intake 35 units (men) or 24 units (women)
- •Current drug abuse
- •Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine
- •Participation in another clinical trial of any investigational drug within 3 months prior to Baseline
- •Other factor that, in the investigator's opinion, excludes the patient from entering the study
研究组 & 干预措施
1
Experimental
Idebenone
干预措施: Idebenone (Drug)
2
Placebo Comparator
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye
时间窗: 24 weeks
次要结局
- Change in the patient's best logMAR visual acuity between baseline and week 24(24 weeks)
- Change in scotoma area in both eyes(Day -1, Week 4, Week 12, Week 24)
- Change in optic nerve fibre layer thickness in both eyes(Day -1, Week 4, Week 12, Week 24)
- Colour contrast sensitivity in both eyes (in a subset of patients)(Day -1, Week 4, Week 12, Week 24)
- logMAR visual acuity as a continuous variable in both eyes(Screening, Day -1, Week 4, Week 12, Week 24, Week 28)
- Clinical Global Impression of Change(Week 4, Week 12 and Week 24)
- Change in Health-Related Quality of Life (HRQOL)(Day -1, Week 4, Week 12, Week 24)
- Change in self-reported general energy levels(Day -1, Week 4, Week 12, Week 24, Week 28)
- Proportion of patients in which visual acuity in the initially least affected eye does not deteriorate to 1.0 log MAR or more ( in LHON patients with eye still less affected than 0.5 logMAR at trial entry)(24 weeks)
- Plasma levels of idebenone matched to measures of efficacy and safety(24 weeks)
- • Best visual acuity at Week 24 (best eye at Week 24) compared to best visual acuity at Baseline (best eye at Baseline)(24 weeks)
- • Count of eyes/ patients for which the visual acuity improves between baseline and week 24(24 weeks)
研究者
研究点 (3)
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