Chicago Sickle Cell Clinical Network

芝加哥镰状细胞临床网络

基本信息

批准号：
7224149
负责人：
JOSEPH DESIMONE
金额：
$ 14.77万
依托单位：
UNIVERSITY OF ILLINOIS AT CHICAGO
依托单位国家：
美国
项目类别：
财政年份：
2006
资助国家：
美国
起止时间：
2006-04-17 至 2011-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7224149
关键词：
Accounting Adult C-reactive protein Cessation of life Chicago Clinical Clinical Markers Clinical Research Clinical Treatment Clinical Trials Coagulation Process Complement Cutaneous Cytochromes Dacogen Data Decitabine Disease Management Dose Double-Blind Method End Point Event Fetal Hemoglobin Fibrin fragment D Frequencies Functional disorder Genes Genetic Hemoglobin Hemolysis Inflammation Injection of therapeutic agent Lactate Dehydrogenase Lactate Dehydrogenases Marrow MeSH Thesaurus Measurement Measures Melanocyte stimulating hormone Mental Depression Morbidity - disease rate Narcotics Normal Statistical Distribution Numbers Oral Oral cavity P-Glycoprotein POMC gene Pain Patients Performance Pharmaceutical Preparations Phase Placebos Pneumonia Population Population Study Purpose Quality of life Randomized Controlled Clinical Trials Rate Research Design Reticulocyte count Review Committee Safety Sample Size Score Severity of illness Sickle Cell Sickle Cell Anemia Standards of Weights and Measures Stroke Sum Symptoms System Testing Therapy Clinical Trials Upper arm Variant Week abstracting acute chest syndrome base clinically significant computerized tools cytotoxic day design experience hydroxyurea mortality mu opioid receptors pill pre-clinical response subcutaneous touchscreen treatment planning trial comparing

项目摘要

DESCRIPTION (provided by applicant): The purpose of this proposal is to participate in the design and performance of multiple multicenter (Clinical Research Network, CRN) therapeutic trials for the treatment of patients with sickle cell disease (SCO). We propose two clinical trials to be considered for performance. 1) To compare dacogen (DAC) to hydroxyurea (HU) in their ability to decrease clinical symptoms of sickle cell disease. Although HU is a benefit in symptomatic SCD, a significant proportion of patients continue to encounter problems despite HU therapy. DAC has been shown to markedly increase HbF levels and decrease surrogate clinical markers of disease severity in all treated patients (HU non-responder and/or intolerant patients). The study design is a phase III, open lable, randomized trial comparing DAC 0.2mg/kg subcutaneously (sq) versus HU 15mg/kg. These doses will be increased until marrow depression occurs. The primary end-point to be compared between the two arms will be crisis frequency. A sample size of 100 patients per arm will provide 90% power to detect a 33% difference in crisis frequency. The purpose of this proposal is to participate in the design and performance of multiple multicenter therapeutic trials for the treatment of patients with sickle cell disease (SCD). 2) To develop an effective pain treatment plan for patients based upon the types of pain they experience., and evaluate them for genotypic variability in a number of polymorphic genes, such as mu receptor, MDR1 protein, and cytochrome 2D6. We will accomplish this by using a reliable touch screen computerized tool that the patient will use to obtain a comprehensive assessment of a patient's pain and their response to treatment. To determine if there is a genetic basis for treatment variation, we will evaluate patients for genotypic variability using a number of polymorphic genes, such as mu receptor, MDR1 protein, and cytochrome 2D6. (End of Abstract)

描述（由申请人提供）：该提案的目的是参与多个多中心（临床研究网络，CRN）治疗镰状细胞病（SCO）患者的治疗试验的设计和实施。我们建议考虑两项临床试验的性能。 1) 比较 dacogen (DAC) 与羟基脲 (HU) 减少镰状细胞病临床症状的能力。尽管 HU 对有症状的 SCD 有好处，但尽管进行了 HU 治疗，仍有相当一部分患者仍遇到问题。 DAC 已被证明可以显着提高所有治疗患者（HU 无反应和/或不耐受患者）的 HbF 水平并降低疾病严重程度的替代临床标志物。该研究设计是一项 III 期、开放标签、随机试验，比较 DAC 0.2mg/kg 皮下注射 (sq) 与 HU 15mg/kg。这些剂量将增加直至发生骨髓抑制。两个组之间比较的主要终点是危机频率。每组 100 名患者的样本量将提供 90% 的功效来检测危机频率 33% 的差异。该提案的目的是参与治疗镰状细胞病（SCD）患者的多个多中心治疗试验的设计和实施。 2) 根据患者所经历的疼痛类型，为患者制定有效的疼痛治疗计划，并评估其许多多态性基因（如 mu 受体、MDR1 蛋白和细胞色素 2D6）的基因型变异。我们将通过使用可靠的触摸屏计算机化工具来实现这一目标，患者将使用该工具来全面评估患者的疼痛及其对治疗的反应。为了确定治疗变异是否存在遗传基础，我们将使用许多多态性基因（例如 mu 受体、MDR1 蛋白和细胞色素 2D6）评估患者的基因型变异。（摘要完）