Phase II Developmental Study on Fatigue in Cancer

描述（由申请人提供）：疲劳是最常见的报告 癌症患者的症状。疲劳的原因是多方面的， 包括疾病本身、抗抑郁治疗、贫血、抑郁症， 营养不良我们的初步数据表明，微量营养素缺乏， 特别是肉毒碱缺乏，可能是疲劳的一个重要因素。 肉毒碱在能量代谢中起着重要作用。全身性衰竭是 其特征在于体重减轻、疲劳、肌肉无力、对 代谢压力和心肌病。我们发现微量营养素缺乏 肉毒碱在17/27例癌症患者中。疲劳和功能障碍的症状 接受口服左旋卡尼汀的患者， 补充。此外，我们在6/10例患者中观察到类似结果， 晚期艾滋病表现为肉毒碱缺乏。 目的：a）确定L-卡尼汀治疗对症状的影响， 患有晚期癌症和血清肉毒碱缺乏的患者的疲劳，和B） 以确定L-肉碱治疗对性能状态，认知功能， 功能、情绪、生活质量和运动活动。 研究设计：我们建议进行一项II期发育随机 双盲安慰剂对照研究，以确定 微量营养素L-肉碱对疲劳和其他结果的患者 晚期癌症130例晚期癌症患者的样本，疲劳和 血清肉毒碱缺乏将被招募到干预研究中。在 第一次就诊时，患者将接受疲劳的基线评估， 体能状态、认知功能、情绪、生活质量和运动 活动a）在第1 - 4周期间，患者将随机接受 左旋肉碱，2克/天，安慰剂。B）在第5 - 8周期间，所有患者将接受 L-肉毒碱的剂量为2克/天，为期4周。措施将 在48小时、2周、4周和8周重复。主要终点将是 4周时疲劳的变化。分析将评估组间差异， 主要终点和其他结局。中期分析将在15 各组均观察4周。配对和非配对数据 小组之间的分析将在一个 统计学家。

DESCRIPTION (provided by applicant): Fatigue is the most frequently reported symptom in patients with cancer. The causes of fatigue are multifactorial and include the disease itself, antineoplastic 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, andcardiomyopathy. We found deficiency of the micronutrient carnitine in 17/27 patients with cancer. Symptoms of fatigue and functional status improved significantly in those patients who received oral L-carnitine supplementation. In addition, we observed similar results in 6/10 patients with end stage AIDS presenting with carnitine deficiency. Objectives: a) To determine the effect of L-carnitine therapy on symptoms of fatigue in patients with terminal cancer 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 Il 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 cancer. A sample of 130 patients with terminal cancer, 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, and quality of life and motor activity. a) During weeks 1-4, patients will be randomized to receive L-carnitine, 2 g/day, placebo. b) During weeks 5-8, all patients will receive L-carnitine at a dose of 2 g/day, for a period of 4 weeks. Measures will be repeated at 48 hs, 2 weeks, 4 weeks and 8 weeks. The primary endpoint will be change in fatigue at 4 weeks. Analysis will evaluate group differences in the primary endpoint and other 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 a statistician.