Pediatric Hydroxyurea Phase III Clinical Trial

目的：本合同的目标是作为儿科羟基脲第三阶段临床试验(婴儿拥抱)的医疗协调中心。临床试验将包括一个医疗协调中心、10个儿科临床中心、一个药房配送中心、一个血液化学实验室和一个细胞遗传学实验室。这项试验是一项随机、双盲的安慰剂对照试验，旨在确定羟基尿素疗法在预防镰状细胞性贫血(HbSS)儿童患者的慢性终末器官损害方面是否有效。医疗协调中心将监督药品分发、中心实验室职能和数据收集。该方案包括一项可行性和安全性试点，从1岁开始，招募了40名儿童。在前40名受试者的数据被dsmb审查后，开始全面招募剩余的样本量。IND编号是2003年4月从食品和药物管理局(FDA)获得的。NHLBI与NICHD根据儿童最佳药品法案(BPCA)建立了合作伙伴关系，以合作进行婴儿拥抱。根据BPCA，如果羟基尿素的有效性和安全性得到证实，NICHD将向FDA提交婴儿拥抱数据集，以便将羟基尿素的标签更改为包括患有镰状细胞性贫血的幼儿。NICHD捐助资金，用于增加新的临床站点作为卫星，研究药物制造和药代动力学分析。

Purpose: The objective of this contract is for the Contractor to serve as the Medical Coordinating Center for the Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG). The clinical trial will include a medical coordinating center, 10 pediatric clinical centers, a pharmacy distribution center, a blood chemistry laboratory, and a cytogenetics laboratory. The trial is a randomized, double blind placebo controlled trial to determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia (HbSS). The medical coordinating center will supervise drug distribution, central laboratory functions, and data collection. The protocol included a Feasibility and Safety pilot beginning at 1 year of age, that recruited 40 children. After the data from the first 40 subjects were reviewed by the DSMB, full recruitment of the remaining sample size began. The IND number was obtained from the Food and Drug Administration (FDA) in April, 2003. NHLBI formed a partnership with NICHD under the Best Pharmaceuticals for Children Act (BPCA) to collaborate on the conduct of BABY HUG. Under the BPCA, if efficacy and safety of hydroxyurea are proven, NICHD will present the BABY HUG data set to the FDA so that the label for hydroxyurea can be changed to include young children with sickle cell anemia. NICHD contributed funds to pay for adding new clinical sites as satellites, study drug manufacturing, and pharmacokinetics analyses.