NCT01962363已完成2 期
A Phase 2A Clinical Trial of EPI-743 (Vincerinone™) on Visual Function in Friedreich's Ataxia Patients With Point Mutations
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
- 4
- 试验地点
- 1
- 主要终点
- Visual function
研究概览
简要总结
The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations
详细描述
The primary objective of this study is to investigate whether treatment with EPI-743 has a discernible impact on visual function-including visual acuity, visual fields and color vision as well as on any of a number of functional and subject/clinician-rated scales relevant in the treatment of Friedreich's ataxia, and to determine the safety of treatment with EPI-743.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of genetically confirmed Friedreich's ataxia point mutation.
- •Visual acuity at baseline more than 15 letters on high contrast EDTRS at four meters.
- •FARS score of 20 to
- •Male or female between 18 and 65 years of age.
- •Agreement to use contraception if within reproductive years
- •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 metropolitan area 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 foods or beverages or bars fortified with Coenzyme Q10, vitamin E, super fortified functional foods or beverages at least 30 days prior to initiation of treatment and for the duration of the study.
- •Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study.
- •Subject can swallow multiple size 0 capsules.
- •Subject has voluntarily signed an IRB approved informed consent form to participate in the study after all relevant aspects of the study have been explained and discussed with the subject.
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.
- •Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening.
- •Clinically significant arrhythmia within past two years requiring treatment.
- •Surgery planned through the duration of the study, including follow-up.
- •Pregnancy or breastfeeding.
- •Anticoagulant therapy within 30 days of enrollment.
研究组 & 干预措施
EPI-743
Experimental
EPI-743, oral, 400mg three times daily for 3 months
干预措施: EPI-743 (Drug)
结局指标
主要结局
Visual function
时间窗: 3 months
Low contrast acuity
Safety parameters
时间窗: 3 months
Clinical and laboratory safety parameters
次要结局
- Health related quality of life(3 months)
- Activities of Daily Living(3 months)
- Cardiac indices(3 months)
- Upper extremity function(3 months)
- Visual function(3 months)
- Neurologic function(3 months)
- Physical function(3 months)
- Disease biomarkers(3 months)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
2 期
A Phase 2A Trial of EPI-743 for Patients With Parkinson's DiseaseParkinson's DiseaseNCT01923584University of South Florida15
已完成
2 期
EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological ImpairmentMethylmalonic Aciduria and Homocystinuria,Cblc TypeRetinopathyGenetic DiseaseNCT01793090Bambino Gesù Hospital and Research Institute30
已完成
4 期
Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia PatientsFriedreich AtaxiaNCT04801303Berta Alemany20
已完成
1 期
Study of FCN-437c in Patients With Advanced Solid TumorsSolid Tumor, AdultNCT03951116Fochon Pharmaceuticals, Ltd.22
已完成
2 期
ENERGI-F703 for Diabetic Foot Ulcers Phase II StudyFoot UlcerWoundsDiabetes MellitusDiabetic FootNCT02672436Energenesis Biomedical Co., Ltd.141