R&D- MEDICAL: BIOMEDICAL (APPLIED RESEARCH/EXPLORATORY DEVELOPMENT)

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• 批准号: 8495479
• 负责人: SOHAIL RANA
• 金额: $ 40.43万
• 依托单位: HOWARD UNIVERSITY
• 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/8495479
• 关键词: Age; Age-Years; Applied Research; Award; Best Pharmaceuticals for Children Act; Brain; Cardiac; Child; Childhood; Chronic; Clinical; Clinical Data; Collaborations; Collection; Contracts; Development; Enrollment; Evaluation; Growth and Development function; Label; Laboratories; Life; Long-Term Effects; Lung; Medical; 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; cohort; design; double-blind placebo controlled trial; follow-up; hydroxyurea; improved; neuropsychological; prevent; research and development; 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.

在2000年，国家心脏，肺和血液研究所（NHLBI）竞争性授予合同，以进行随机的，双盲的，安慰剂对照的试验，对镰状细胞病（SCD）进行测试，以测试羟基脲（HU）的假设，即在招募两个年龄的儿童之前，可以防止hydroxyurea（HU）防止慢性结束器官损害。该研究还旨在监测研究治疗，评估生长和发育的临床反应能力，并从研究治疗中监测毒性。该试验从2003年10月至2007年6月招募了193名SCD的受试者，年龄在9至18个月之间。受试者仍在研究药物两年。这种续签的目的是对入学的儿童III期临床试验（BABY HOUG）的儿童进行结构化的长期临床，影像学和实验室随访，并在生命的前十年进行了后续研究I。此外，后续研究II将在长期使用过程中评估羟基脲对器官功能或毒性的影响。 根据《最佳儿童法》（BPCA）NHLBI，作为IND赞助商，与NICHD合作，将向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。