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: 6768461
• 负责人: JOHN L THOMPSON
• 金额: $ 28.61万
• 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/6768461
• 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（由申请方提供）：QALS-CLIN和QALS-STAT是两个独立但高度协调的单位，共同构成QALS，这是一项辅酶Q10与安慰剂比较的两阶段、II期、随机、安慰剂对照、双盲、多中心临床试验（目标入组18个临床研究中心的185例患者）。 第一阶段确定两种剂量的辅酶Q10中的哪一种是优选的（1,000 mg）。或2,000毫克。每天），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疗效证据证明进行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修订版（ALSFRSr）评分从基线（随机化）到9个月的变化，初步研究已证实其可作为生存金标准的替代品。 还测试了四个预先指定的次要支持假设。 基于ALSFRSr子评分的替代结局指标可能被证明更敏感，将在三级分析中作为未来试验的候选指标进行探索。 QALS-STAT的目标是：1）尽量减少所需的随访时间和患者数量。 2)进行、有效整合并负责所有QALS数据管理和统计操作。 3)及时向临床协调中心（CCC）传输QALS CLIN的主要研究者履行其职责所需的所有数据，以确保患者安全并指导所有临床操作。 目标1是通过选择程序和使用替代终点来实现的，这将随访时间缩短了50%。 目标2由统计分析中心实现，该中心的工作人员在协调、数据管理和临床试验编程以及其统计要求方面具有丰富的经验。 目标3由QALS-CLIN和QALS-STAT调查员和高级协调员组成的业务委员会确保，该委员会根据严格规定和控制的通信协议经常开会。