AWARD OF BABY HUG FOLLOW UP STUDY II CLINICAL SITE.

荣获婴儿拥抱后续研究 II 临床网站奖。

• 批准号: 8495478
• 负责人: SHERRON JACKSON
• 金额: $ 26.84万
• 依托单位: MEDICAL UNIVERSITY OF SOUTH CAROLINA
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-01-10 至 2016-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8495478
• 关键词: Age; Age-Years; Award; Best Pharmaceuticals for Children Act; Brain; Cardiac; Child; Childhood; Chronic; Clinical; Clinical Data; Collaborations; Collection; Contracts; Enrollment; Evaluation; Follow-Up Studies; Growth and Development function; Label; Laboratories; Life; Long-Term Effects; Lung; Monitor; National Heart, Lung, and Blood Institute; National Institute of Child Health and Human Development; Natural History; Organ; Pharmaceutical Preparations; Phase III Clinical Trials; Randomized; Recruitment Activity; Risk; Sickle Cell Anemia; Structure; Testing; Toxic effect; United States Food and Drug Administration; clinical research site; cohort; design; double-blind placebo controlled trial; follow-up; hydroxyurea; improved; neuropsychological; prevent; standard of care

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.

2000年，美国国家心脏、肺和血液研究所（NHLBI）竞争性授予合同，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，以检验羟基脲（HU）可以预防两岁前招募的儿童发生慢性终末器官损伤的假设。本研究还旨在监测对研究治疗的临床反应，评估生长和发育，并监测研究治疗的毒性。该试验从2003年10月至2007年6月入组了193例年龄在9至18个月之间的SCD受试者。受试者继续接受研究药物治疗2年。本次更新的目的是对入组原始儿科羟基脲III期临床试验（BABY HUG）和随访研究I的儿童进行结构化的长期临床、影像学和实验室随访，随访至出生后10年。此外，随访研究II将提供长期使用期间对器官功能或毒性的影响的评价。 根据《儿童最佳药物法案》（BPCA），NHLBI与NICHD合作，作为IND申办者，将向美国食品药品监督管理局（FDA）提交临床数据，以寻求HU的儿科使用标签。