Phase II Developmental Study on Fatigue in AIDS Patients

描述(由申请人提供)：疲劳是一种常见的症状 艾滋病患者。疲劳的原因是多方面的，包括 疾病本身、抗病毒治疗、贫血、抑郁和营养不良。我们的 初步数据表明，微量营养素缺乏，特别是 肉碱缺乏，可能是疲劳的重要因素。肉毒碱起到了 在能量代谢中起主要作用。全身衰竭的特征是体重 丧失，疲劳，肌肉无力，对代谢应激的耐受性下降，以及 心肌病。我们发现有6/10的人微量营养素缺乏。 终末期艾滋病患者出现犬类缺乏症。症状： 这些患者的疲劳和功能状态显著改善 口服L-卡尼汀补充剂。 目的：1)探讨L-卡尼汀治疗慢性阻塞性肺疾病的临床疗效。 晚期艾滋病和血清肉碱缺乏患者的疲劳感；和b) 确定L-卡尼汀治疗对运动状态、认知功能的影响 这些患者的功能、情绪、生活质量和运动能力。 研究设计：我们建议进行二期发育随机双盲 确定微量营养素效果的安慰剂对照研究 L-卡尼汀对艾滋病晚期患者疲劳等结局的影响。一个 44例晚期艾滋病、疲劳和血肉碱患者的样本 将缺陷性纳入干预研究。在第一次访问时， 患者将接受疲劳、表现状态、 认知功能、情绪、生活质量和运动活动。A)在数周内 1-4，患者将随机接受L-卡尼汀，3g/天，或安慰剂治疗。b) 在5-8周期间，所有患者将接受L-卡尼汀3g/d治疗1周。 疗程4周。主要终点是4周后的疲劳感改变。 分析将评估两组在主要终点和结果方面的差异。 一旦观察到15名患者，将进行中期分析 4周时分组。组之间的配对和非配对数据分析将 在统计学家的协助下进行的。

DESCRIPTION (provided by applicant): Fatigue is a commonly reported symptom in patients with AIDS. The causes of fatigue are multifactorial and include the disease itself, antiviral therapies, anemia, depression and malnutrition. Our preliminary data suggest that micronutrient deficiencies, specifically carnitine deficiency, may be an important factor in fatigue. Carnitine plays a major role in energy metabolism. Systemic depletion is characterized by weight loss, fatigue, muscle weakness, decreased tolerance to metabolic stress and cardiomyopathy. WE found deficiency of the micronutrient carnitine in 6/10 patients with end stage AIDS presenting with canitine deficiency. Symptoms of fatigue and functional status improved significantly in those patients who received oral L-carnitine supplementation. Objectives: a) to determine the effect of L-carnitine therapy on symptoms of fatigue in patients with terminal AIDS and serum carnitine deficiency; and b) to determine the effect of L-carnitine therapy on performance status, cognitive function, mood, quality of life, and motor activity in these patients. Study Design: We propose to conduct a Phase II developmental randomized double-blind placebo-controlled study to determine the effect of the micronutrient L-carnitine on fatigue and other outcomes in patients with terminal AIDS. A sample of 44 patients with terminal AIDS, fatigue and serum carnitine deficiency will be recruited into the intervention study. At the first visit, the patients will receive a baseline assessment of fatigue, performance status, cognitive function, mood, quality of life, and motor activity. a) During weeks 1-4, patients will be randomized to receive L-carnitine, 3g/day, or placebo. b) During weeks 5-8, all patients will receive L-carnitine dose at 3 g/day for a period of 4 weeks. The primary endpoint will be change in fatigue at 4 weeks. Analysis will evaluate group differences in the primary endpoint and outcomes. An interim analysis will be done once 15 patients have been observed in each group at 4 weeks. Paired and unpaired data analyses between groups will be conducted with the assistance of the statistician.