A Phase II, Open Label Prospective Single Center Drug Study Evaluating the Safety and Efficacy of (+)-Epicatechin in Subjects With Friedreich's Ataxia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Change in Ventricular Hypertrophy as Shown on Cardiac MRI
研究概览
简要总结
This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
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
- 10 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of Friedreich's Ataxia (FA) by Frataxin genetic testing and/or Frataxin enzyme analysis
- •Between age 10 and 50 years of age, inclusive
- •Body weight of 25 kilograms or higher
- •Minimum of one affected organ (cardiac or neurological) system, as evidenced by clinical signs/symptoms
- •Disease duration ≤7 years, based on onset date of FA symptoms
- •Has no known contraindication to gadolinium contrast such as severe allergy or Glomerular Filtration Rate <30 ml/min/m^
- •Has no known contraindication to non-contrast Magnetic Resonance Imaging (MRI) evaluation such as pacemaker or magnetically active metal fragments.
- •Women of childbearing age must:
- •Have a negative pregnancy human chorionic gonadotropin test prior to receiving study drug.
- •Agree to use contraception for the duration of the study drug dosing, plus 1 month after completion of the study.
排除标准
- •Advanced cardiac failure, New York Heart Association (NYHA) Classification Scale-Class IV (advanced stage heart failure)
- •Clinically significant comorbidities that may also lead to cardiomyopathy, for example long standing hypertension, familial cardiomyopathy.
- •Clinically significant comorbidities that would, in the opinion of the investigators, compromise the interpretation of test results.
- •Pregnant, breast-feeding or planning to become pregnant during study timeframe.
- •Patients with contraindications to regadenoson, i. e. second- or third-degree atrioventricular (AV) block or sinus node dysfunction. Has received an investigational drug within thirty (30) days of baseline visit.
- •Thrombocytopenia (<125 x 10^9/Liter) or prolonged Prothrombin Time/Partial Thromboplastin Time (PT/PTT) at baseline.
- •Clinically significant hypotension (systolic blood pressure <90) due to heart failure or other conditions.
研究组 & 干预措施
(+)-Epicatechin
Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks
干预措施: (+)-Epicatechin (Drug)
结局指标
主要结局
Change in Ventricular Hypertrophy as Shown on Cardiac MRI
时间窗: Baseline, 24 weeks
Left ventricular mass and left ventricular (LV) mass indexed to body surface area estimated by Left Ventricle (LV)cavity dimension and wall thickness at end-diastole. Normal values of LV mass indexed to body surface area are found to be 49-115 gL/m2 in men and 43-95 g/m2 in women.
Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score
时间窗: Baseline, 24 weeks
The Friedreich Ataxia Rating Scale (FARS) is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate (assesses speech speed by repetitions of pronouncing "PaTa" ) and low-contrast letter acuity. FARS is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate and low-contrast letter acuity. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.
次要结局
未报告次要终点
研究者
Ralitza Gavrilova
MD, Sponsor-Investigator
Mayo Clinic