Pediatric Hydroxyurea Phase III Clinical Trial

儿科羟基脲III期临床试验

• 批准号: 8328449
• 负责人: LINDA MONUM
• 金额: $ 67.17万
• 依托单位: CLINICAL TRIALS AND SURVEYS CORPORATION
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-08-01 至 2012-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8328449
• 关键词: 1 year old; Adverse effects; Best Pharmaceuticals for Children Act; Blood Chemical Analysis; Child; Childhood; Chronic; Clinical; Clinical Trials; Contractor; Contracts; Cytogenetics; Data; Data Collection; Data Set; Drug Kinetics; Fetal Hemoglobin; Funding; Growth and Development function; Incidence; Label; Laboratories; Medical; National Heart, Lung, and Blood Institute; National Institute of Child Health and Human Development; Organ; Patients; Pharmaceutical Preparations; Pharmacy Distributions; Phase III Clinical Trials; Prevention; Protocols documentation; Randomized; Recruitment Activity; Safety; Sample Size; Sickle Cell Anemia; Toxic effect; United States Food and Drug Administration; clinical research site; double-blind placebo controlled trial; drug distribution; hydroxyurea; indexing

Purpose: The objective of this contract is for the Contractor to serve as the Medical Coordinating 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 medical coordinating center will supervise drug distribution, central laboratory functions, and data collection. 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 new clinical sites as satellites, study drug manufacturing, and pharmacokinetics analyses.

目的：本合同的目的是让承包商担任小儿羟基脲III期临床试验（BABY HUG）的医学协调中心。临床试验将包括一个医疗协调中心，10个儿科临床中心，一个药房配送中心，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、双盲安慰剂对照试验，旨在确定羟基脲治疗是否能有效预防镰状细胞贫血（HbSS）儿科患者的慢性终末器官损伤。医疗协调中心将监督药物分配、中心实验室功能和数据收集。该方案包括从1岁开始的可行性和安全性试点，招募了40名儿童。在DSMB审查了前40例受试者的数据后，开始招募剩余样本量。IND编号于2003年4月从美国食品药品监督管理局（FDA）获得。NHLBI根据《儿童最佳药物法》（BPCA）与NICHD建立了伙伴关系，合作开展BABY HUG。根据BPCA，如果证明了羟基脲的有效性和安全性，NICHD将向FDA提交BABY HUG数据集，以便可以将羟基脲的标签更改为包括患有镰状细胞贫血的幼儿。NICHD出资支付增加新的临床研究中心作为卫星、研究药物生产和药代动力学分析的费用。