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

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 胰岛素样生长因子I(IGF-I)是对垂体生长激素(GH)的反应而产生的，是生长的主要调节因子。一些儿童患有IGF-I缺乏症，因为他们不能对自己的生长激素做出反应。他们有严重的生长迟缓，对传统疗法没有反应。本研究评价重组人胰岛素样生长因子-I(rhIGF-I)治疗矮小儿童生长激素不敏感的安全性和有效性。儿童必须有临床证明的生长激素不敏感，血清IGF-I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，或在生长激素治疗期间产生生长激素中和抗体。一旦入选，儿童每天两次皮下注射重组人胰岛素样生长因子-I，直到他们达到最终身高。主要疗效终点是生长速度。受试者至少每6个月接受一次评估。安全措施包括监测血液化学、肾和脾生长以及心功能。

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. GInsulin-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.