GH IN CHILDREN WITH SHORT STATURE DUE TO GROWTH HORMONE INSENSITIVITY (GHIS)

因生长激素不敏感而导致身材矮小的儿童的 GH (GHIS)

• 批准号: 7374484
• 负责人: STEVEN D CHERNAUSEK
• 金额: $ 1.06万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 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/7374484
• 关键词: GH; CHILDREN; SHORT; STATURE; DUE

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. Insulin-like growth factor I (IGF-I) is produced in response to pituitary growth hormone (GH) and is a major regulator of growth. Some children have IGF-I deficiency because they cannot respond to their own GH. They have severe growth retardation unresponsive to conventional therapies. This study evaluates the safety and efficacy of recombinant human IGF-I (rhIGF-I) given to short children with growth hormone insensitivity. Children must have clinically documented GH insensitivity with serum IGF-I levels that are low for age, normal GH secretion and evidence for failure of GH action, or have developed GH-neutralizing antibodies during GH treatment. Once enrolled, children are treated with subcutaneous injection of rhIGF-I twice daily until they reach final height. The primary efficacy endpoint is growth rate. Subjects are evaluated a minimum of every 6 months. Safety measures include monitoring of blood chemistries, kidney and spleen growth, and cardiac function.

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。胰岛素样生长因子I （IGF-I）是在垂体生长激素（GH）的作用下产生的，是生长的主要调节因子。有些儿童患有igf - 1缺乏症，因为他们对自身的生长激素没有反应。他们有严重的生长迟缓，对传统疗法没有反应。本研究评估了重组人IGF-I （rhIGF-I）用于生长激素不敏感的矮个子儿童的安全性和有效性。儿童必须有临床证明的生长激素不敏感，血清igf - 1水平低于年龄，生长激素分泌正常，有生长激素作用失败的证据，或者在生长激素治疗期间产生了生长激素中和抗体。一旦入组，儿童每天两次皮下注射rhigf - 1，直到他们达到最终身高。主要疗效终点是生长速率。受试者至少每6个月评估一次。安全措施包括监测血液化学、肾脏和脾脏的生长以及心脏功能。