A Multi-Center, Double-Blind, Randomized, Parallel Group Tolerability Study of Coenzyme Q10 (UbiquinonE)in PRE-manifest Huntington's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 90
- 试验地点
- 13
- 主要终点
- Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment.
研究概览
简要总结
To establish the tolerability of treatment with 600, 1200 or 2400 mg per day of coenzyme Q10 in pre-manifest participants carrying the CAGn expansion for Huntington's Disease (HD).
详细描述
secondary objectives:
- To assess the change from baseline to 20 weeks on biomarkers of oxidative stress (8OHdG and 8OHrG) and DNA repair mechanisms (OGG1) in pre-manifest participants treated with 600, 1200 or 2400 mg per day of CoQ10.
- To assess the dose-response relationship between CoQ10 at dosages of 600, 1200 or 2400 mg per day and 8OHdG/8OHrG and OGG1.
- To assess the serum levels of CoQ10 at 600, 1200 or 2400 mg in pre-manifest participants and their relationship to 8OHdG/8OHrG and OGG1.
- To assess the feasibility of implementing a preventive therapeutic trial in a pre-manifest population.
- To assess the utility and stability of clinical measures of HD, social relations, behavior and employment in a pre-manifest sample enrolled in a treatment trial.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants will be positive for the CAGn expansion in the Huntingtin gene (>36 repeats) and be pre-manifest by virtue of scoring 3 or less on diagnostic confidence level (Question 17 of the UHDRS)
- •Participants will have received genetic testing prior to enrollment through a standard pre-manifest testing protocol.
- •18 years of age or older.
- •Concomitant medications are permitted with the exception of CoQ10, creatine > 5g/day and warfarin.
排除标准
- •History of intolerability to CoQ
- •CoQ10 use within 60 days prior to randomization.
- •Unstable medical or psychiatric illness;
- •Substance abuse within one year of the baseline visit.
- •Pregnancy, breastfeeding or lack of reliable contraception in women of childbearing age.
- •Subjects with known allergy to FD&C #6 yellow food coloring.
研究组 & 干预措施
600 mg per day of CoQ10
All participants will start on a dosage of 600 mg/day of CoQ10 in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20.
干预措施: CoQ10 (Drug)
1200 mg per day of CoQ10
All participants will start on a dosage of 600 mg/day in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20.
干预措施: CoQ10 (Drug)
2400 mg per day of CoQ10
All participants will start on a dosage of 600 mg/day in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20.
干预措施: CoQ10 (Drug)
结局指标
主要结局
Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment.
时间窗: 20 weeks
No dosage modifications, reported as a %
次要结局
- CoQ10 Levels(change from baseline to 20 weeks)
- 8OHdG Levels(change from baseline to 20 weeks)