PEDIATRIC HYDROXYUREA PHASE III CLINICAL TRIAL: BABY HUG

儿科羟基脲 III 期临床试验：婴儿拥抱

• 批准号: 7376189
• 负责人: CATERINA MINNITI
• 金额: $ 6.09万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-12-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7376189
• 关键词: PEDIATRIC; HYDROXYUREA; PHASE; III; CLINICAL

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Hydroxyurea (HU) has demonstrated laboratory and clinical efficacy for adults with sickle cell anemia (SCA). Pediatric patients with SCA also benefit from HU therapy. Although HU therapy has emerged as an exciting and efficacious therapeutic agent for patients with SCA, important issues remain regarding its use, especially for those less than 2 years old. The primary objective of this study is to determine whether daily oral hydroxyurea can reduce by ¿50% chronic organ damage that develops in young children with SCA. Secondary objectives of this study include: 1) to determine the relationship between fetal hemoglobin levels and chronic organ damage in young children with SCA and 2) investigate the safety of HU for young children with SCA regarding a) physical growth and development, b) neuropsychological development, c) immunological responses, and d) mutagenic effects on DNA.

该主题项目是利用NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子弹和调查员（PI）可能已经从其他NIH来源获得了主要资金，因此可以在其他清晰的条目中代表。列出的机构是针对该中心的，这不是调查人员的机构。羟基脲（HU）证明了镰状细胞贫血（SCA）的成年人的实验室和临床效率。 SCA的儿科患者也受益于HU疗法。尽管HU疗法已成为SCA患者的激动人心，有效的治疗剂，但有关其使用的重要问题，尤其是对于不到2年的人而言。这项研究的主要目的是确定每日口腔羟基脲是否可以减少50％的慢性器官损伤，而SCA患者会导致幼儿产生。这项研究的次要目标包括：1）确定SCA幼儿胎儿血红蛋白水平与慢性器官损害之间的关系； 2）研究SCA幼儿在a）a）身体生长和发育方面的HU安全性，b）b）神经心理学发展，c）免疫反应，c）免疫反应，d）d）对DNA的杂种作用。