NCT01728064已完成2 期
Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia
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
- 60
- 试验地点
- 3
- 主要终点
- Visual Function
研究概览
简要总结
The purpose of this study is to examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.
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
- 18 Years 至 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of genetically confirmed Friedreich's ataxia
- •Visual acuity at baseline more than 15 letters on EDTRS at four meters
- •FARS score of 20 to 90
- •Agreement to use contraception if within reproductive years (see specifics in section D1, page 21)
- •Hormone replacement therapy, if used, must remain stable for the duration of the study
- •Willingness and ability to comply with study procedures
- •Willingness and ability to arrive at study site day prior to evaluations
- •Abstention from use of dietary supplements and non-prescribed medications at least 30 days prior to initiation of treatment and for the duration of the study. This would specifically include idebenone, Coenzyme Q10 and vitamin E
- •Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study
排除标准
- •Allergy to EPI-743 or sesame oil or nuts
- •Clinically significant bleeding condition or abnormal PT/PTT INR (INR > two; PTT > two-times normal)
- •Liver insufficiency with LFTs greater than three-times upper normal limit at screening
- •Renal insufficiency with creatinine > 1.5 at screening
- •Fat malabsorption syndromes
- •Any other respiratory chain diseases of the mitochondria or inborn errors of metabolism
- •Any other ophthalmologic conditions
- •History of alcohol or drug abuse
- •Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening
- •Clinically significant arrhythmia within past two years requiring treatment
- •Anticoagulant therapy within 30 days of enrollment.
研究组 & 干预措施
Placebo
Placebo Comparator
Placebo capsules three times daily
干预措施: Placebo (Drug)
EPI-743 400 mg
Active Comparator
EPI-743 at a dose of 400 mg three times daily
干预措施: EPI-743 400 mg (Drug)
EPI-743 200 mg
Active Comparator
EPI-743 at a dose of 200 mg three times daily
干预措施: EPI-743 200 mg (Drug)
结局指标
主要结局
Visual Function
Low contrast visual acuity
次要结局
- Disease biomarkers
- Cardiac function
- Disease improvement
- Quality of life
- Color vision
- Neurologic function
- Neuromuscular function
- Safety
- Visual Function(Baseline, Months 3, 6, 9 and 12)
研究者
研究点 (3)
Loading locations...
相似试验
进行中（未招募）
不适用
Evaluation of safety and efficacy on visual acuity outcome of intravitreal somministration of Bevacizumab in patients with diabetic retinopathy - NDMedDRA version: 8.1Level: LLTClassification code 10012689Term: Diabetic retinopathyDiabetic RetinopathyEUCTR2006-005315-10-ITOSPEDALE SACRO CUORE-DON CALABRIA
进行中（未招募）
不适用
Evaluation of Safety and Efficacy on Visual Acuity Outcome and Choroidal Neovascularization of Intravitreal Somministration of Bevacizumab Avastin, Roche, United Kingdom in patients with Subfoveal Choroidal Neovascularization due to Age Related Macular Degeneration - NDSubfoveal Choroidal Neovascularization due to Age Related Macular Degeneration who did not respond to currently available treatmentsMedDRA version: 6.1Level: PTClassification code 10060823EUCTR2006-002424-42-ITOSPEDALE SACRO CUORE-DON CALABRIA150
招募中
不适用
Healthy Little EyesNeonatal EncephalopathyEncephalopathyHypoxic-Ischemic EncephalopathyNCT05971446University of Wisconsin, Madison125
已完成
不适用
A Study to Evaluate the BrainPort® Vision Device in Subjects Who Are BlindBlindnessNCT01488786Wicab75
Unknown
不适用
Prospective Clinical Trials to Assess Performance and Safety of EyeSwift ProVisual FunctionsNCT05382676NovaSight500