L2762G EFF OF NUTROPIN AQ FOR TRMT OF GROWTH RESTRICTION IN CHILD W CF

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。Nutroin AQ是一种重组人生长激素(RhGH)。重组人生长激素用于治疗生长受限已在其他慢性疾病中得到证实。使用重组人生长激素治疗可以显著改善生长和瘦体重。有一些小型研究表明，重组人生长激素可以改善肺功能中的用力肺活量。囊性纤维化儿童的典型特征是线形生长减少，这预示着肺功能的下降。囊性纤维化的生长受限是多因素的，但可能是由于饮食摄入不足和代谢需求增加所致。与正常人群相比，充足的营养摄入并没有改善囊性纤维化患者的生长。这项拟议的研究是一项II期多中心、随机、对照、开放标签的研究，目的是探讨重组人生长激素治疗患有囊性纤维化的生长受限儿童的安全性和有效性。本研究旨在确定努托品AQ对囊性纤维化儿童的生长受限、瘦体重和肺功能的影响。安全参数将是血糖水平的测量、肺功能测试、血清化学变化、对重组人生长激素的免疫反应以及受试者报告或观察到的症状。疗效将包括身高和体重的测量、肺功能测试和双能X射线吸收法(DEXA)扫描。

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. Nutropin AQ is a recombinant human growth hormone (rhGH). The use of rhGH for the treatment of growth restriction has been demonstrated in other chronic diseases. Treatment with rhGH can result in significant improvement in growth and lean body mass. There are some small studies which have shown that rHGH may improve the forced vital capacity in pulmonary function. Children with Cystic Fibrosis characteristically have reduced linear growth, which is predictive of declines in pulmonary function. The restricted growth in Cystic Fibrosis is multi-factorial, but probably due to a combination of poor dietary intake and increased metabolic demands. Adequate nutritional intake has not improved growth in Cystic Fibrosis patients when compared to a normal population. The proposed study is a phase II, multicenter, randomized, controlled, open-label study of the safety and efficacy of using rhGH in growth restricted children with Cystic Fibrosis. The study is designed to determine the effect of Nutropin AQ on growth restriction, lean body mass and pulmonary function in children with Cystic Fibrosis. Safety parameters will be a measurement of blood glucose levels, pulmonary function testing, and any changes in serum chemistries, immunologic response to the rhGH, as well as reported or observed symptoms of subjects. Efficacy will be a measurement of height and weight, pulmonary function testing, and dual energy x-ray absorptiometry (DEXA) scans.