Marian Anderson Sickle Cell Center-Sickle Cell Disease Clinical Research Network

描述（由申请人提供）： 该临床中心将为圣克里斯托弗儿童医院（SCHC）、托马斯杰斐逊大学医院（TJUH）、科赛尔儿童医院（KCH）和杜邦儿童医院（DCH）的962名SCD患者提供最先进的临床研究活动支持。其中三个研究中心（SCHC、KCH、TJUH）目前是Marian安德森综合（MAC）镰状细胞中心的一部分，目前在综合中心申办的几项I/II/III期中心间研究中负责招募，也是在Dampier博士和Rockstein女士的指导下通过MAC中心的临床核心进行的。St. Christopher's Hospital的中心PI和中心首席临床协调员均获得了临床研究专业人员协会的认证（分别为临床试验研究者和CRA认证），并拥有多年成功协调和监督各中心研究活动的记录。拟议的网络临床试验解决了SCD患者临床护理中的重要问题。第一项研究是头痛管理的第四阶段，将建立在描述性和流行病学数据的基础上，这些数据是综合中心网络的C数据项目的一部分。本研究提出了一项为期4个月的安慰剂对照RCT，用于预防性治疗（阿米替林，有或没有CBT训练）在7- 17岁的儿童中进行，在前3个月内每月头痛>/= 2次，而伴随研究将评估阿米替林或双丙戊酸钠ER在4个月双盲平行治疗中减少复发性头痛天数的有效性，在每月偏头痛≥ 2次的成人中进行安慰剂对照试验。第二项临床试验将检查两种替代吗啡PCA给药方案与标准PCA治疗相比的疗效。它还将确定吗啡药代动力学的变化、由药物遗传学确定的吗啡药效学的变化或受试者心理社会因素是否改变各种PCA方案的功效。还提出了一个结局核心，其重点是开发各种结局指标和描述性临床信息，以支持对镰状细胞相关疼痛治疗进行适当的高质量临床试验。 这些研究的表现将促进镰状细胞相关疼痛及其后果的临床理解。他们的结果将刺激镰状疼痛的创新临床试验的发展。(End摘要）

DESCRIPTION (provided by applicant): This Clinical Center will provide support for state-of-the-art clinical research activities for 962 SCD patients at St. Christopher's Hospital for Children (SCHC), Thomas Jefferson University Hospital (TJUH), Kosair Children's Hospital (KCH), and duPont Children's Hospital (DCH). Three of these sites (SCHC, KCH, TJUH) are currently part of the Marian Anderson Comprehensive (MAC) Sickle Cell Center and currently lead enrollment in several of the phase l/ll/lll Inter-Center studies sponsored by the Comprehensive Centers, also under the direction of Dr. Dampier and Ms. Rockstein through the MAC Center's Clinical Core. The Center PI and Center Chief Clinical coordinator at St. Christopher's Hospital are both credentialed by the Association of Clinical Research Professionals (Clinical Trial Investigator, and CRA certifications respectively) and have a several year record of successful coordination and supervision of research activities at the various Center sites. The proposed network clinical trials address important issues in clinical care of SCD patients. The first study is a phase IV of headache management that will build on descriptive and epidemiologic data being gathered as part of the C-data project of the Comprehensive Center's Network. This study proposes a 4 month placebo controlled RCT of prophylactic therapy (amitryptline with or without CBT training) in children aged 7- 17 years with >/= 2 headaches per month in the preceding 3 months, while a companion study will evaluate the effectiveness of amitriptyline or divalproex ER in reducing the number of days of recurrent headaches in a 4 month double blind, parallel treatment, placebo-controlled trial of adults with >/= 2 migraine headaches per month. The second clinical trial will examine the efficacy of two alternative morphine PCA dosing regimens in comparison to a standard PCA therapy. It will also determine whether variations in morphine pharmacokinetics, in morphine pharmacodynamics as determined by pharmacogenetics, or subject psychosocial factors, alter the efficacy of the various PCA regimens. An Outcomes Core is also proposed which will focus on developing a variety of outcome measures and descriptive clinical information to support the appropriate, conduct of high quality clinical trials of therapies for sickle cell-related pain. The performance of these studies will advance the clinical understanding of sickle cell-related pain and its consequences. Their results will stimulate the development of innovative clinical trials of sickle pain. (End of abstract)