BABY HUG FOLLOW-UP STUDY II

2000年，美国国家心肺和血液研究所(NHLBI)竞争性地授予合同，在患有镰状细胞病(SCD)的幼儿中进行一项随机、双盲、安慰剂对照试验，以测试羟基尿素(HU)可以防止两岁前招募的儿童发生慢性终末器官损害的假设。这项研究还旨在监测临床对研究治疗的反应性，评估生长发育，并监测研究治疗的毒性。从2003年10月到2007年6月，这项试验招募了193名9到18个月大的SCD受试者。受试者在为期两年的时间里继续服用研究药物。此次更新的目的是对参加最初的儿科羟基尿素第三阶段临床试验(婴儿拥抱)和I期后续研究的儿童进行系统的长期临床、放射学和实验室随访，直至生命的第一个十年。此外，后续研究II将评估羟基尿素在长期使用过程中对器官功能或毒性的影响。 根据儿童最佳药物法案(BPCA)，NHLBI作为IND赞助商，将与NICHD合作，向食品和药物管理局(FDA)提交临床数据，以寻求HU的儿科使用标签。

In 2000, the National Heart, Lung, and Blood Institute (NHLBI) competitively awarded contracts to conduct a randomized, double-blind, placebo-controlled trial in young children with sickle cell disease (SCD) to test the hypothesis that Hydroxyurea (HU) can prevent the onset of chronic end organ damage in children recruited before two years of age. The study was also designed to monitor clinical responsiveness to study treatments, to assess growth and development, and to monitor for toxicity from study treatments. The trial enrolled 193 subjects with SCD between the ages of 9 and 18 months from October 2003 to June 2007. Subjects remained on study drug for a period of two years. The purpose of this renewal is to perform structured long-term clinical, radiographic, and laboratory follow-up of children enrolled in the original Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) and the Follow-Up Study I through the first decade of life. In addition, the Follow-Up Study II will provide evaluation of hydroxyurea effects on organ function or toxicity during long term use. In collaboration with the NICHD under the Best Pharmaceuticals for Children Act (BPCA) NHLBI, as the IND sponsor, will submit clinical data to the Food and Drug Administration (FDA) to seek pediatric use labeling for HU.