PEDIATRIC HYDROXYUREA PHASE III CLINICAL TRIAL

BABY HUG是一项随机、双盲、安慰剂对照试验，旨在确定羟基脲是否可以预防镰状细胞性贫血幼儿慢性终末器官损伤的发生。BABY HUG是在成功完成另外两项镰状细胞病试验之后进行的，即羟基脲治疗镰状细胞性贫血（MSH）的多中心研究和儿科羟基脲（PED HUG）试验。将招募约200名6个月至24个月的镰状细胞病患儿，接受羟脲或安慰剂治疗。在试验开始时，将对儿童进行筛查，以检查脑、肾、肺和脾功能异常的迹象以及发育里程碑。然后他们将被随机分配接受羟基脲或安慰剂，随后每年对主要器官系统的慢性终末器官损伤进行研究。

BABY HUG is a randomized, double-blind, placebo-controlled trial to determine if hydroxyurea can prevent the onset of chronic end organ damage in young children with sickle cell anemia. BABY HUG follows the successful completion of two other sickle cell disease trials, the Multicenter Study of Hydroxyurea in Sickle Cell Anemia (MSH) and the Pediatric Hydroxyurea (PED HUG) Trial. Approximately 200 children, ages six months to 24 months, with sickle cell disease will be recruited to receive either hydroxyurea or placebo. The children will be screened at trial start-up for signs of abnormal brain, renal, pulmonary, and splenic function, and for developmental milestones. They will then be randomly assigned to receive hydroxyurea or a placebo and followed with yearly studies of chronic end organ damage of the major organ systems.