Phase II Developmental Study on Fatigue in Cancer

描述(由申请人提供)：最常报告的是疲劳 癌症患者的症状。疲劳的原因是多方面的，而且 包括疾病本身、抗肿瘤治疗、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， 具体地说，肉碱缺乏可能是疲劳的一个重要因素。 肉碱在能量代谢中起着重要作用。全身性衰竭是 以体重减轻、疲劳、肌肉无力、耐力下降为特征 代谢应激和心肌病。我们发现微量营养素缺乏 27例癌症患者中有17例使用肉碱。疲劳和功能障碍症状 口服L-卡尼汀的患者病情明显改善 补充。此外，我们在6/10的患者中观察到了类似的结果 以肉碱缺乏为表现的终末期艾滋病。 目的：1)探讨L肉碱治疗对晚期癌症伴血清肉碱缺乏患者乏力症状的影响。 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Il期随机发育试验。 双盲安慰剂对照研究 微量营养素L-卡尼汀对慢性阻塞性肺疾病患者疲劳等结局的影响 晚期癌症。130例晚期癌症患者的样本，疲倦和 将血肉碱缺乏纳入干预研究。在… 第一次就诊时，患者将接受疲劳的基线评估， 表现状态、认知功能、情绪、生活质量和运动 活动。A)在1-4周内，患者将随机接受 L-卡尼汀，2g/天，安慰剂。B)在5-8周内，所有患者将接受 L-卡尼汀，2g/d，疗程4周。措施将是 分别于48hs、2周、4周、8周重复。主端点将是 疲劳在4周时的变化。分析将评估群体差异在 主要终点和其他结果。中期分析将在15分钟内完成 每组均于4周时观察患者情况。配对数据和未配对数据 小组之间的分析将在一名 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.