A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, 28 Day, Three-arm, Parallel Group Study of A0001 in the Treatment of Subjects With Friedreich's Ataxia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 42
- 试验地点
- 1
- 主要终点
- Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test
研究概览
简要总结
This is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to receive either a high dose of A0001, a low dose of A0001 or placebo for 28 days.
Eligible subjects will return within 21 days of screening for the baseline visit and randomization to one of three potential treatments. The subjects will be required to take 3 capsules of study medication in the morning with a morning meal and 3 capsules of study medication at night with an evening meal for 28 days. Additional visits to the clinic are planned for Day 14 and Day 28, at which time a number of clinical and biochemical assessments will be done.
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 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals with genetically confirmed Friedreich's Ataxia (GAA or point mutation)
- •Impaired Glucose Tolerance, measured by Oral GTT
排除标准
- •Overt Diabetes Mellitus
- •Presence of clinically significant cardiovascular disease
研究组 & 干预措施
Placebo
Placebo
干预措施: placebo (Drug)
High Dose
A0001 (0.75 g BID)
干预措施: alpha-tocopherolquinone (A0001) (Drug)
Low Dose
A0001 (0.5 g BID)
干预措施: alpha-tocopherolquinone (A0001) (Drug)
结局指标
主要结局
Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test
时间窗: Baseline, Day 14 and Day 28
次要结局
- FARS/neurological exam(Baseline and Day 28)
- Global Impression of Clinical Severity(Baseline, Day 14 and Day 28)
- HbA1C(Baseline, Day 14 and Day 28)
- Plasma 1,5-anhydroglucitol (1,5-AG) (Glycomark)(Baseline, Day 14 and Day28)
- Timed 25 Foot Walk Test(Baseline and Day 28)
- 9-Hole Peg Test(Baseline, Day 14 and Day 28)
- Activities of Daily Living(Baseline and Day 28)
- SF-36®(Baseline and Day 28)
- Specific Activity of Complex 1 in whole blood(Baseline, Day 14 and Day 28)
- Sensitivity index (SI) calculated from IVGTT(Baseline, Day 14 and Day 28)
- Glucose effectiveness (SG) calculated from IVGTT(Baseline, Day 14 and Day 28)
- AIRg for glucose and insulin during IVGTT(Baseline, Day 14 and Day 28)
- Fasting Glucose, Insulin and Lactate(Baseline, Day 14 and Day 28)
- Vision Low Contrast Letter Acuity Test(Baseline and Day 28)
- Modified Fatigue Impact Scale(Baseline and Day 28)