Pediatric Hydroxyurea Phase III Clinical Trial

儿科羟基脲III期临床试验

基本信息

批准号：
7657727
负责人：
金额：
$ 7.42万
依托单位：
SUNY DOWNSTATE MEDICAL CENTER
依托单位国家：
美国
项目类别：
财政年份：
资助国家：
美国
起止时间：
至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/7657727
关键词：
Pediatric Hydroxyurea Phase III Clinical

项目摘要

Purpose: The objective of this contract is for the Contractor to serve as a clinical 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 clinical center is responsible for recruitment, enrollment, and patient follow-up examinations for the purpose of monitoring clinical responsiveness to study treatments, to assess growth and development, and to monitor for toxicity to study treatments. 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

目的：该合同的目的是使承包商成为小儿羟基脲III期临床试验（Baby Hug）的临床中心。临床试验将包括一个医疗协调中心，10个儿科临床中心，药房分配中心，一个血液化学实验室和一个细胞遗传学实验室。该试验是一项随机的双盲安慰剂对照试验，以确定羟基脲治疗是否有效预防镰状细胞贫血（HBSS）的小儿患者的慢性终端器官损伤。临床中心负责招募，入学和患者随访检查，目的是监测研究治疗，评估生长和发育以及监测研究治疗的毒性的临床反应。该协议包括从1岁开始的可行性和安全飞行员，招募了40名儿童。 DSMB审查了前40名受试者的数据后，开始全面招募样本量。 IND编号是从2003年4月从食品药品监督管理局（FDA）获得的。NHLBI根据《最佳儿童法案》（BPCA）与NICHD建立了合作伙伴关系（BPCA），以协作婴儿拥抱的行为。在BPCA下，如果证明了羟基脲的功效和安全性，NICHD将向FDA展示婴儿拥抱数据集，以便可以更改羟基脲的标签，以包括患有镰状细胞贫血的幼儿。 NICHD为增加