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。其中三个站点(SCHC、KCH、TJUH)目前是玛丽安·安德森综合(MAC)镰刀细胞中心的一部分，目前牵头参加由综合中心赞助的多项L/11/11期中心间研究，该研究也由丹皮尔博士和Rockstein女士通过MAC中心的临床核心进行指导。圣克里斯托弗医院的PI中心和中心首席临床协调员都获得了临床研究专业人员协会的认证(分别是临床试验调查员和CRA认证)，并在各中心站点成功协调和监督研究活动方面有多年的成功记录。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，它将建立在作为综合中心网络C-Data项目的一部分收集的描述性和流行病学数据的基础上。这项研究提出了一项为期4个月的安慰剂对照随机对照试验，对7-17岁的患有头痛的儿童进行为期4个月的预防治疗(接受或不接受CBT训练)，同时一项配套研究将在4个月的双盲、平行治疗、安慰剂对照试验中评估阿米替林或双丙戊酸ER在减少头痛复发天数方面的有效性。第二项临床试验将检验两种替代吗啡PCA剂量方案的疗效，并与标准的PCA疗法进行比较。它还将确定吗啡药代动力学的变化，药物遗传学确定的吗啡药效变化，或主观心理社会因素是否会改变各种PCA方案的疗效。还提出了一个结果核心，它将专注于开发各种结果衡量标准和描述性临床信息，以支持进行适当的、高质量的镰状细胞相关疼痛治疗的临床试验。 这些研究的实施将促进对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刀痛的创新临床试验的发展。(摘要结束)

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)