Clinical Trial of High Dose CoQ10 in ALS-QALS-=STAT

高剂量 CoQ10 在 ALS-QALS-=STAT 中的临床试验

• 批准号: 6874859
• 负责人: JOHN L THOMPSON
• 金额: $ 24.53万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-04-01 至 2008-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6874859
• 关键词: amyotrophic lateral sclerosis; biomedical resource; clinical trial phase II; cofactor; cooperative study; data management; health science research; patient care management; patient safety /medical error; statistical service /center; ubiquinone

DESCRIPTION (provided by applicant): QALS-CLIN and QALS-STAT are two independent, but highly coordinated units, which together constitute QALS, a two-stage, phase II, randomized, placebo-controlled, double-blind, multi-center clinical trial of Coenzyme Q10 against placebo (target enrollment 185 patients at 18 clinical sites). The first stage identifies, which of two doses of CoQ10 is preferred (1,000 mg. or 2,000 mg. daily), using a selection procedure to hold the number of patients required to a minimum. The second stage compares the preferred dose against placebo to assess whether there is sufficient evidence of efficacy to justify proceeding to a phase III trial. The primary outcome measure is the change in Amyotrophic Lateral Sclerosis Functional Rating Scale revised (ALSFRSr) score from baseline (randomization) to 9 months, which preliminary research has validated as a surrogate for the gold standard of survival. Four pre-specified secondary supporting hypotheses are also tested. Alternative outcome measures, based on ALSFRSr subscores that may prove more sensitive, will be explored in tertiary analyses as candidates for future trials. The goals of QALS-STAT are: 1) To minimize follow-up-time and number of patients required. 2) To conduct, efficiently integrate, and be responsible for all QALS data management and statistical operations. 3) To transmit to the Clinical Coordinating Center (CCC) in a timely fashion all of the data which the principal investigator of QALS CLIN needs to meet her responsibilities, in terms of ensuring patient safety and directing all clinical operations. Goal 1 is accomplished by the selection procedure, and the use of a surrogate endpoint, which cuts follow-up time by 50%. Goal 2 is achieved by a Statistical Analysis Center, whose personnel have extensive experience in coordination, data management, and programming for clinical trials, as well as their statistical requirements. Goal 3 is ensured by an Operations Committee of QALS-CLIN and QALS-STAT investigators and senior coordinators, which meets frequently under tightly specified and controlled communication protocols.

描述（由申请人提供）：QALS- clini和QALS- stat是两个独立但高度协调的单元，它们共同构成了QALS，一个两阶段，II期，随机，安慰剂对照，双盲，多中心的辅酶Q10对抗安慰剂的临床试验（目标入组185名患者，18个临床站点）。第一阶段确定两种辅酶q10剂量中的哪一种是优选的（每天1000毫克或2000毫克），使用选择程序将所需的患者数量保持在最低限度。第二阶段将首选剂量与安慰剂进行比较，以评估是否有足够的证据证明其有效性，以证明进行III期试验是合理的。主要结局指标是肌萎缩侧索硬化症功能评定量表（ALSFRSr）评分从基线（随机化）到9个月的变化，初步研究已证实该评分可作为生存金标准的替代指标。还检验了四个预先指定的次要支持假设。基于可能更敏感的ALSFRSr评分的替代结果测量将在三级分析中作为未来试验的候选指标进行探索。